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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F8232f1f8c3437335788aafde7f218b09b936e0d0-2912x1632.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F8232f1f8c3437335788aafde7f218b09b936e0d0-2912x1632.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F8232f1f8c3437335788aafde7f218b09b936e0d0-2912x1632.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F8232f1f8c3437335788aafde7f218b09b936e0d0-2912x1632.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F8232f1f8c3437335788aafde7f218b09b936e0d0-2912x1632.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F8232f1f8c3437335788aafde7f218b09b936e0d0-2912x1632.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-contain" loading="lazy"/></noscript></span></a></div><div class="border-t-gray-100 border-t-2 flex-auto py-2 "><span class="text-sm text-gray-500 py-2">November 21st 2024</span><div class="py-3"><a class="lg:text-[20px] text-[19px] font-[500]" href="/view/neuronal-deficit-in-severe-infantile-sma-broader-than-previously-thought-study-finds">Neuronal Deficit in Severe Infantile SMA Broader Than Previously Thought, Study Finds </a></div><div class="pb-2"><div><span class="text-md "><span class="mr-1 italic">By </span><a class="mr-1 text-sky-800 hover:text-primary" href="/authors/jared-kaltwasser"><i>Jared Kaltwasser</i></a></span></div></div><a href="/view/neuronal-deficit-in-severe-infantile-sma-broader-than-previously-thought-study-finds"><a href="/view/neuronal-deficit-in-severe-infantile-sma-broader-than-previously-thought-study-finds"><p class="mt-1 text-gray-800 text-[13px] line-clamp-6 text-hidden">New research suggests previous assumptions about the pathogenesis of spinal muscular atrophy (SMA) are inadequate. </p></a></a></div></div><div class="flex-wrap w-[60%] flex "><a class="flex w-[50%] flex-col sm:pl-[35px] pl-[20px] pb-[35px] " href="/view/low-bone-mineral-density-risk-increases-with-age-in-sma"><div class="w-full lg:h-[134px] h-[100px] relative bg-default-logo-background "><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="" 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src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F926a727a9b1c24ca69f6188f75edc552caeed107-5824x3264.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="lg:object-cover object-contain " loading="lazy"/></noscript></span></div><div class="border-t-gray-100 border-t-2 pt-2 pb-2 flex-auto "><a href="/view/low-bone-mineral-density-risk-increases-with-age-in-sma"><span class="text-sm text-gray-500">November 13th 2024</span></a><div><a class="text-[14px]" href="/view/low-bone-mineral-density-risk-increases-with-age-in-sma"><p class="line-clamp-4 text-hidden font-[500]">Low Bone Mineral Density Risk Increases With Age in SMA</p></a></div><div class="py-2"><div><span class="text-md "><span class="mr-1 italic">By </span><a 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class="jsx-1434825611 text-[18px] font-bold text-[#000000] text-left line-clamp-3 text-hidden">Dr Jill Jarecki Discusses the Early SMA Treatment for Infants</p></div></div></div></a><a target="" style="scroll-snap-align:center;text-decoration:none" class="jsx-4064126826 sm:w-[280px] xs:w-[240px] w-[65%] h-auto flex flex-col space-y-3 flex-none select-none no-underline" href="/view/drs-mary-schroth-and-jill-jarecki-discuss-treatments-for-all-sma-types"><div class="jsx-4064126826 w-full h-full flex flex-col overflow-hidden relative bg-white text-center"><div class="jsx-4064126826 relative w-full shadow-md shadow-grey-800 aspect-video"><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="fill" class="object-cover" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%"/><noscript><img alt="" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, 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src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-cover" loading="lazy"/></noscript></span></div><div class="jsx-4064126826 content-wrap"><span class="jsx-4064126826 py-3 text-sm text-gray-500 text-left"><i class="jsx-4064126826">October 18th 2020</i></span><div class="jsx-4064126826 w-full h-fit mb-4"><p class="jsx-4064126826 text-[18px] font-bold text-[#000000] text-left line-clamp-3 text-hidden">Drs Mary Schroth and Jill Jarecki Discuss Treatments for All SMA Types</p></div></div></div></a><a target="" style="scroll-snap-align:center;text-decoration:none" class="jsx-187450181 sm:w-[280px] 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srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F1c26cd7f0f594f71d84aebeb4fa427a48f1ad4a3-1280x720.png%2FScreen%2520Shot%25202020-10-09%2520at%25204.21.06%2520PM%25202.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F1c26cd7f0f594f71d84aebeb4fa427a48f1ad4a3-1280x720.png%2FScreen%2520Shot%25202020-10-09%2520at%25204.21.06%2520PM%25202.png%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F1c26cd7f0f594f71d84aebeb4fa427a48f1ad4a3-1280x720.png%2FScreen%2520Shot%25202020-10-09%2520at%25204.21.06%2520PM%25202.png%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F1c26cd7f0f594f71d84aebeb4fa427a48f1ad4a3-1280x720.png%2FScreen%2520Shot%25202020-10-09%2520at%25204.21.06%2520PM%25202.png%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, 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src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F1c26cd7f0f594f71d84aebeb4fa427a48f1ad4a3-1280x720.png%2FScreen%2520Shot%25202020-10-09%2520at%25204.21.06%2520PM%25202.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-cover" loading="lazy"/></noscript></span></div><div class="jsx-187450181 content-wrap"><span class="jsx-187450181 py-3 text-sm text-gray-500 text-left"><i class="jsx-187450181">October 11th 2020</i></span><div class="jsx-187450181 w-full h-fit mb-4"><p class="jsx-187450181 text-[18px] font-bold text-[#000000] text-left line-clamp-3 text-hidden">Dr Jill Jarecki Discusses the Current Pipeline for SMA Therapies</p></div></div></div></a><a target="" style="scroll-snap-align:center;text-decoration:none" class="jsx-609564708 sm:w-[280px] xs:w-[240px] w-[65%] h-auto flex flex-col space-y-3 flex-none select-none no-underline" href="/view/dr-mary-schroth-discusses-at-home-treatment-options-for-patients-with-sma"><div class="jsx-609564708 w-full h-full flex flex-col overflow-hidden relative bg-white text-center"><div class="jsx-609564708 relative w-full shadow-md shadow-grey-800 aspect-video"><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="fill" class="object-cover" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%"/><noscript><img alt="" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720.png%2FMarySchroth.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-cover" loading="lazy"/></noscript></span></div><div class="jsx-609564708 content-wrap"><span class="jsx-609564708 py-3 text-sm text-gray-500 text-left"><i class="jsx-609564708">October 4th 2020</i></span><div class="jsx-609564708 w-full h-fit mb-4"><p class="jsx-609564708 text-[18px] font-bold text-[#000000] text-left line-clamp-3 text-hidden">Dr Mary Schroth Discusses At-Home Treatment Options for Patients With SMA</p></div></div></div></a></div></div></div><div class="flex items-center space-x-4 border-0 border-b border-secondary select-none py-1"><h2 class="text-3xl text-primary">More News</h2></div><div class="flex flex-wrap w-full pb-4"><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-1"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/muscle-directed-therapy-apitegromab-meets-primary-end-point-in-phase-3-sapphire-trial-of-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/3b8a9306ddc33b9690a5368b3412e8df33debd09-5906x3898.jpg?fit=crop&auto=format" alt="SMA puzzle pieces | image credit: Dzmitry - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 17th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/muscle-directed-therapy-apitegromab-meets-primary-end-point-in-phase-3-sapphire-trial-of-sma">Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/marco-meglio">Marco Meglio</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/muscle-directed-therapy-apitegromab-meets-primary-end-point-in-phase-3-sapphire-trial-of-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Patients aged 2-21 receiving apitegromab showed clinically meaningful motor function improvements, with a favorable safety profile consistent with long-term data, as Scholar Rock prepares for US and EU regulatory submissions in Q1 2025.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/breaking-novel-prognostic-biomarkers-discovered-for-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d3eea8b1d68140d8cbdd9ad2d33f356568e44816-5376x3584.jpg?fit=crop&auto=format" alt="Biomarker identification has the potential to transform screening, early identification and treatment for diseases like SMA | image credit: Катерина Євтехова - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 9th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/breaking-novel-prognostic-biomarkers-discovered-for-sma">Novel Prognostic Biomarkers Discovered for SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/karen-jacobson-sive">Karen Jacobson-Sive</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/breaking-novel-prognostic-biomarkers-discovered-for-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">CNTN1 and NRXN3 clearly differentiated severe and milder forms of spinal muscular atrophy (SMA). </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/5-year-review-further-bolsters-support-for-newborn-sma-screening"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/46cc98cba7554bb9d19fe855d6658fad88698ea8-5760x3034.jpg?fit=crop&auto=format" alt="Newborn screening can provide numerous benefits for infants who may have unknown conditions or complications | image credit: Peakstock - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 3rd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/5-year-review-further-bolsters-support-for-newborn-sma-screening">5-Year Review Further Bolsters Support for Newborn SMA Screening </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/5-year-review-further-bolsters-support-for-newborn-sma-screening"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A retrospective analysis on newborn screening for spinal muscular atrophy (SMA) provides further evidence for the benefits of early identification and treatment of the disease. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/new-research-unearths-evidence-of-metabolic-disruption-in-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/6445313cb23a06f6cad709653e7d5c8fdf4b78c3-5000x3250.jpg?fit=crop&auto=format" alt="SMA has previously been associated with multiple metabolic and nutritional issues | image credit: JEGAS RA - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 21st 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/new-research-unearths-evidence-of-metabolic-disruption-in-sma">New Research Unearths Evidence of Global Metabolic Disruption in Patients With SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/new-research-unearths-evidence-of-metabolic-disruption-in-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A recent analysis investigated the state of metabolic disruption experienced by patients with spinal muscular atrophy (SMA), adding to the literature on nutritional and metabolic complications linked to SMA. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/decision-making-in-sma-complicated-by-insufficient-treatment-comparison"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/e32d368ab659149e3f5832f8d1ee9c58ea889e90-5376x3584.jpg?fit=crop&auto=format" alt="Clinical decision-making in SMA could benefit from more comprehensive trial data comparing SMA treatments | image credit: HASAN - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 19th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/decision-making-in-sma-complicated-by-insufficient-treatment-comparison">Decision-Making in SMA Complicated by Insufficient Treatment Comparison</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/nameera-temkar-md">Nameera Temkar, MD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/decision-making-in-sma-complicated-by-insufficient-treatment-comparison"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Clinical decision-making can be difficult for patients with SMA due to the lack of clinical trials comparing treatment options. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/higher-dose-nusinersen-meets-primary-end-point-in-phase-2-3-devote-study-for-spinal-muscular-atrophy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/567de38f26ce65e5f995fdab0a6990db7a0b9b02-3508x2480.jpg?fit=crop&auto=format" alt="Nusinersen was the first FDA-approved drug for the treatment of SMA in pediatric and adult patients in December 2016 | image credit: AGPhotography - stock.adobe.come" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 17th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/higher-dose-nusinersen-meets-primary-end-point-in-phase-2-3-devote-study-for-spinal-muscular-atrophy">Higher-Dose Nusinersen Meets Primary End Point in Phase 2/3 DEVOTE Study for Spinal Muscular Atrophy</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/isabella-ciccone-mph">Isabella Ciccone, MPH</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/higher-dose-nusinersen-meets-primary-end-point-in-phase-2-3-devote-study-for-spinal-muscular-atrophy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Based on the positive topline data, the company plans to submit for regulatory approval for a higher dose of nusinersen for spinal muscular atrophy (SMA) treatment.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-7"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/fatiguability-in-sma-may-benefit-from-nusinersen-but-further-research-is-needed"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d8de599bce63e2591c72b91f754893b502b21685-5250x3500.jpg?fit=crop&auto=format" alt="The 6-Minute Walking Test is a common tool for measuring fatiguability in SMA | image credit: Antonioguillem - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 28th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/fatiguability-in-sma-may-benefit-from-nusinersen-but-further-research-is-needed">Nusinersen May Improve Fatiguability in SMA, but More Research Is Needed </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/fatiguability-in-sma-may-benefit-from-nusinersen-but-further-research-is-needed"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Findings from an Italian cohort suggest that nusinersen may benefit fatigue measures in patients with spinal muscular atrophy (SMA) type III; however, drawing definitive conclusions in this area remains difficult. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/smn-depletion-may-increase-risk-of-liver-damage-fatty-liver-in-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/ea840df9cead0ba4acb662a60d766b97f74cf9d0-4200x2700.jpg?fit=crop&auto=format" alt="The SMN protein is expressed thoughout many body tissues types, including the liver | image credit: Rasi - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 22nd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/smn-depletion-may-increase-risk-of-liver-damage-fatty-liver-in-sma">SMN Depletion May Increase Risk of Liver Damage, Fatty Liver in SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/smn-depletion-may-increase-risk-of-liver-damage-fatty-liver-in-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A retrospective, single-center cohort study utilized liver ultrasound, serum collection, and proteome analysis to draw associations between spinal motor neuron (SMN) protein depletion and heightened risk for fatty liver disease in spinal muscular atrophy (SMA). </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/virtual-reality-is-a-game-changer-during-blood-draws-for-pediatric-patients-with-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/59c533995a73aa1f3c94a2a54a665dfd69dab299-5477x2739.jpg?fit=crop&auto=format" alt="VR can reduce fear and anxiety associated with drawing blood in patients with SMA | image credit: matrosovv - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 21st 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/virtual-reality-is-a-game-changer-during-blood-draws-for-pediatric-patients-with-sma">Virtual Reality Is a Game-Changer During Blood Draws for Pediatric Patients With SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/abraham-homer-ms">Abraham Homer, MS</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/virtual-reality-is-a-game-changer-during-blood-draws-for-pediatric-patients-with-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">In honor of Spinal Muscular Atrophy (SMA) Awareness Month, Abraham Homer, MS, the gaming technology supervisor and creative technologist at Children's Hospital Colorado, shared how virtual reality transformed care for patients with SMA.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/new-motor-milestones-achieved-or-sustained-with-apitegromab-therapy-in-non-ambulatory-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/a642a49a556ba21dd35f8cd1718ce085123a954f-5163x2905.jpg?fit=crop&auto=format" alt="Apitegromab administered at 20mg/kg every 4 weeks lead to improvements in motor functions in patients with SMA | image credit: Justlight - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 5th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/new-motor-milestones-achieved-or-sustained-with-apitegromab-therapy-in-non-ambulatory-sma">New Motor Milestones Achieved or Sustained With Apitegromab in Nonambulatory SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/new-motor-milestones-achieved-or-sustained-with-apitegromab-therapy-in-non-ambulatory-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The extension of the TOPAZ study provided support for the long-term benefits and safety of apitegromab therapy in patients with spinal muscular atrophy (SMA) at 36 months. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/potential-biomarkers-predict-disease-severity-and-treatment-response-in-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/c1d414e04045697dbdc36f1594c777e582892f76-1200x800.jpg?fit=crop&auto=format" alt="SMA spinal muscular atrophy | Image Credit: © Rochu_2008 - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 26th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/potential-biomarkers-predict-disease-severity-and-treatment-response-in-sma">Potential Biomarkers Predict Disease Severity and Treatment Response in SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/potential-biomarkers-predict-disease-severity-and-treatment-response-in-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Results from a single-center, observational, prospective study suggested that inflammatory markers could predict disease severity and treatment efficacy for patients with spinal muscular atrophy (SMA). </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/sma-birth-prevalence-in-us-lower-than-expected-new-report-finds"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/8b9195e98cf6fccba0be11f9f3f9e719a70dc670-6048x4032.jpg?fit=crop&auto=format" alt="Globally, SMA is diagnosed for an estimated 1 in every 10,000 births | image credit: Alexander Raths - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 19th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/sma-birth-prevalence-in-us-lower-than-expected-new-report-finds">SMA Birth Prevalence in US Lower Than Expected, New Report Finds</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/sma-birth-prevalence-in-us-lower-than-expected-new-report-finds"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A new report provided updated figures estimated the birth prevalence of spinal muscular atrophy (SMA) in the US. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-13"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/dual-therapy-well-tolerated-but-presents-little-benefit-to-infantile-onset-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/2de8278ba38f5b7bd4b37044c3661ff70e4e1ae2-5000x2363.jpg?fit=crop&auto=format" alt="Onasemnogene abeparvovec is a gene therapy approved in the last decade for SMA | image credit: ipopba - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">Published: July 12th 2024 | Updated: July 12th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/dual-therapy-well-tolerated-but-presents-little-benefit-to-infantile-onset-sma">Dual Therapy Well-Tolerated but Presents Little Benefit to Infantile-Onset SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/dual-therapy-well-tolerated-but-presents-little-benefit-to-infantile-onset-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A multicenter pilot study concluded that dual therapy in spinal muscular atrophy (SMA) does not provide further benefits to infants at risk for SMA type 1. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/final-readout-of-firefish-study-showcases-risdiplam-s-pronounced-effects-in-spinal-muscular-atrophy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/66c94cf99b709474dd3b4c3e1b26aecec7b34c92-6720x2880.jpg?fit=crop&auto=format" alt="Risdiplam demonstrated great 5-year efficacy for improving motor functioning and feeding capabilities in children with type I SMA | image credit: Sittipol - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 3rd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/final-readout-of-firefish-study-showcases-risdiplam-s-pronounced-effects-in-spinal-muscular-atrophy">Final Readout of FIREFISH Study Showcases Risdiplam’s Pronounced Effects in Spinal Muscular Atrophy</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/marco-meglio">Marco Meglio</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/final-readout-of-firefish-study-showcases-risdiplam-s-pronounced-effects-in-spinal-muscular-atrophy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The FIREFISH study showed that most children with type I spinal muscular atrophy (SMA) maintained or improved motor functions and feeding abilities over 5 years when treated with risdiplam.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/qualitative-study-explores-caregiver-experiences-in-sma-treatment"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/86dd6d493a2e8985d74713f4e5ff7c43297f682b-7934x5292.jpg?fit=crop&auto=format" alt="Medical Caregiver Concept | image credit: Alexis Scholtz - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">December 31st 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/qualitative-study-explores-caregiver-experiences-in-sma-treatment">Qualitative Study Explores Caregiver Experiences in SMA Treatment</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/qualitative-study-explores-caregiver-experiences-in-sma-treatment"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">This qualitative study conducted interviews with caregivers of patients with spinal muscular atrophy (SMA) to explore their unique experiences and challenges in navigating health systems to access disease-modifying therapies. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/more-expensive-sma-medication-use-led-to-lower-health-care-resource-utilization"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/66c1591b2bef5d67825d9c88b2fdb7bf9429f3d2-4577x2647.jpg?fit=crop&auto=format" alt="hospital | Image credit: VILevi - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">December 27th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/more-expensive-sma-medication-use-led-to-lower-health-care-resource-utilization">More-Expensive SMA Medication Use Led to Lower Health Care Resource Utilization</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/skylar-jeremias">Skylar Jeremias</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/more-expensive-sma-medication-use-led-to-lower-health-care-resource-utilization"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Patients with spinal muscular atrophy (SMA) treated with a more expensive medication were found to have higher pharmacy costs but lower SMA-related health care resource utilization and medical costs compared with patients receiving standard-of-care nusinersen monotherapy.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/onasemnogene-abeparvovec-effective-in-treatment-of-older-heavier-children-with-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/e43167f246727ae6a96a64e67a48ed31585b9839-6720x4480.jpg?fit=crop&auto=format" alt="Heavier Baby | image credit: Leka - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">December 26th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/onasemnogene-abeparvovec-effective-in-treatment-of-older-heavier-children-with-sma">Onasemnogene Abeparvovec Effective in Treatment of Older, Heavier Children with SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/onasemnogene-abeparvovec-effective-in-treatment-of-older-heavier-children-with-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/alternative-method-of-nusinersen-administration-identified-for-sma-cases-with-spinal-deformities"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/c53dc9cdac5dafecb4c926067d979f0b8bb7ad44-3600x2400.jpg?fit=crop&auto=format" alt="Lumbar Region Model | image credit: Crystal light - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">December 20th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/alternative-method-of-nusinersen-administration-identified-for-sma-cases-with-spinal-deformities">Alternative Method of Nusinersen Administration Identified for SMA Cases With Spinal Deformities</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/alternative-method-of-nusinersen-administration-identified-for-sma-cases-with-spinal-deformities"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A retrospective, single-center study out of the Czech Republic tested the safety and efficacy of unilateral interlaminar fenestration (UILF) on the convex side in cases of SMA that also exhibit severe spinal deformities. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-19"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/top-5-most-read-sma-content-2023"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/72b4a0733f36413ab54280a85487ee24de1adb09-3187x1625.png?fit=crop&auto=format" alt="Top 5 SMA Content 2023" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">December 16th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/top-5-most-read-sma-content-2023">Top 5 Most-Read Spinal Muscular Atrophy Content 2023</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/top-5-most-read-sma-content-2023"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">This year’s most-read spinal muscular atrophy(SMA) articles explored mental health, new technologies, caregiver well-being, and more. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/acetaminophen-use-requires-careful-dosing-in-patients-with-sma-study-says"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/fe283a2d3e21b36ebe89c1c3ae7a9636dbe6584e-2800x1868.jpg?fit=crop&auto=format" alt="Acetaminophen Bottle | image credit: luchschenF - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">December 6th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/acetaminophen-use-requires-careful-dosing-in-patients-with-sma-study-says">Acetaminophen Use Requires Careful Dosing in Patients With SMA, Study Says</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/acetaminophen-use-requires-careful-dosing-in-patients-with-sma-study-says"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A non-randomized, open-label, single-site clinical trial evaluated the safety and pharmacokinetics of acetaminophen intake in adults and children with spinal muscular atrophy (SMA) type II.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/sma-may-be-linked-to-cognitive-impairment-in-chinese-patients"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/624b54d83c3bcbdcd3d504ac7f1c494ba3a101a8-4608x3072.jpg?fit=crop&auto=format" alt="Cognitive Impairment Model | image credit: Pol Solé - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">November 30th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/sma-may-be-linked-to-cognitive-impairment-in-chinese-patients">SMA May Be Linked to Cognitive Impairment in Chinese Patients</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/sma-may-be-linked-to-cognitive-impairment-in-chinese-patients"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A cross-sectional study suggests that spinal muscular atrophy (SMA) can contribute to cognitive impairment. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/innovations-in-sma-treatment-the-last-decade-and-beyond"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/c85890cc9e676682277141f2ef5f2b981c1fbc87-5906x3898.jpg?fit=crop&auto=format" alt=" SMA Puzzle | image credit: Dzmitry - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">November 21st 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/innovations-in-sma-treatment-the-last-decade-and-beyond">Innovations in SMA Treatment: The Last Decade and Beyond</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/innovations-in-sma-treatment-the-last-decade-and-beyond"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The recent availability of 3 FDA-approved treatments for patients with spinal muscular atrophy (SMA) has dramatically altered patient outcomes and provided a wealth of new avenues for SMA research. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/q-a-lingering-theories-and-questions-in-sma-research"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/7419bbf3e0f6d9c8184f0a771cea5608edc0a073-1000x563.jpg?fit=crop&auto=format" alt="Alexander Fay, MD, PhD, UCSF Benioff Children's Hospital" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">November 18th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/q-a-lingering-theories-and-questions-in-sma-research">Q&A: Lingering Theories and Questions in SMA Research</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/q-a-lingering-theories-and-questions-in-sma-research"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Alexander Fay, MD, PhD, joined for a Q&A about the current state of spinal muscular atrophy (SMA) treatment and the unanswered questions in the field. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/researchers-identify-potential-biomarkers-for-sma-motor-improvement-following-nusinersen-treatment"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/9de3528b1e21d800d6422121e419f7fcd8a6429a-5000x3750.jpg?fit=crop&auto=format" alt="Biomarker Concept | image credit: molekuul.be - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">November 17th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/researchers-identify-potential-biomarkers-for-sma-motor-improvement-following-nusinersen-treatment">Researchers Identify Potential Biomarkers for SMA Motor Improvement Following Nusinersen Treatment </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/researchers-identify-potential-biomarkers-for-sma-motor-improvement-following-nusinersen-treatment"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A retrospective, exploratory analysis identifies biomarkers predictive of spinal muscular atrophy (SMA) response to nusinersen and suggests the efficacy of using machine learning algorithms to anticipate patient outcomes.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-25"></div></div></div><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/type-1-sma-linked-to-increased-risk-of-nephrolithiasis-among-children"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/1b4cabc2aa4eb7c5816f2bdeef83e40781daea40-6720x4480.jpg?fit=crop&auto=format" alt="SMA | Image credit: Rochu_2008 – stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">November 16th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/type-1-sma-linked-to-increased-risk-of-nephrolithiasis-among-children">Type 1 SMA Linked to Increased Risk of Nephrolithiasis Among Children</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/hayden-e-klein">Hayden E. Klein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/type-1-sma-linked-to-increased-risk-of-nephrolithiasis-among-children"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">According to researchers, the most prevalent risk factors in children with type 1 spinal muscular atrophy (SMA) are hypercalciuria and elevated urine specific gravity.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/rainbowfish-results-highlight-risdiplam-as-effective-treatment-for-presymptomatic-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/4b44d8f9157d0bec5df24a015a988c7bf1f523da-6720x4480.jpg?fit=crop&auto=format" alt="Infant Sitting on its Own | image credit: Prostock-studio - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 27th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/rainbowfish-results-highlight-risdiplam-as-effective-treatment-for-presymptomatic-sma">RAINBOWFISH Results Highlight Risdiplam as Effective Treatment for Presymptomatic SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/marco-meglio">Marco Meglio</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/rainbowfish-results-highlight-risdiplam-as-effective-treatment-for-presymptomatic-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">After 1 year of treatment with risdiplam, 81% of the 26-infant cohort was able to sit independently for 30 seconds, a milestone not typically reached in spinal muscular atrophy (SMA).</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/novel-screening-method-shows-promise-in-detecting-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/34add436adfb7e033ededa058234d8231a2f3a66-5394x3596.jpg?fit=crop&auto=format" alt="PCR loading | image credit: angellodeco - stock.adobe.come" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 26th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/novel-screening-method-shows-promise-in-detecting-sma">Novel Screening Method Shows Promise in Detecting SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/novel-screening-method-shows-promise-in-detecting-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Researchers have developed a new line method (NLM) that showcased benefits for genetic screening in spinal muscular atrophy (SMA). </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/diaphragm-ultrasound-imaging-showcases-promise-for-respiratory-assessments-in-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/2bd451b150475476b34e1bade4d458f98a49b9ef-3840x2160.jpg?fit=crop&auto=format" alt="Human Diaphragm | Image credit: magicmine - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 12th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/diaphragm-ultrasound-imaging-showcases-promise-for-respiratory-assessments-in-sma">Diaphragm Ultrasound Imaging Showcases Promise for Respiratory Assessments in SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/diaphragm-ultrasound-imaging-showcases-promise-for-respiratory-assessments-in-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">In this prospective study, respiratory monitoring of patients with spinal muscular atrophy (SMA) benefited from the use of diaphragm ultrasound imaging. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/study-validates-the-positive-effects-of-nusinersen-on-bone-health-in-patients-with-sma-type-2-and-3"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d90aa9719f2a92603268e787a52563656ff1696d-4006x3072.jpg?fit=crop&auto=format" alt="Bone Deterioration | freshidea - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 5th 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/study-validates-the-positive-effects-of-nusinersen-on-bone-health-in-patients-with-sma-type-2-and-3">Study Validates the Positive Effects of Nusinersen on Bone Health in Patients With SMA Types 2 and 3</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kyle-munz">Kyle Munz</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/study-validates-the-positive-effects-of-nusinersen-on-bone-health-in-patients-with-sma-type-2-and-3"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">In a multidisciplinary follow-up study, researchers analyzed bone health decline and the effects of nusinersen in patients with spinal muscular atrophy (SMA) types 2 and 3.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/patterns-of-lung-function-decline-likely-not-predictive-of-respiratory-failure-in-sma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/9944c90056bb126ca0f4e85f5b38c0f8bd5fa7b0-1280x853.jpg?fit=crop&auto=format" alt="Image of a mechanical ventilator: sudok1 - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">March 21st 2023</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/patterns-of-lung-function-decline-likely-not-predictive-of-respiratory-failure-in-sma">Patterns of Lung Function Decline Likely Not Predictive of Respiratory Failure in SMA</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jaime-rosenberg">Jaime Rosenberg</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/patterns-of-lung-function-decline-likely-not-predictive-of-respiratory-failure-in-sma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Following the course of lung function in treatment-naïve patients, researchers found that lung function decline does not accelerate prior to mechanical ventilation.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div></div><div class="w-full text-center flex justify-center pb-24"><a class="px-4 py-2 border-y border-r bg-primary text-white" href="/compendium/sma?page=1">1</a><a class="px-4 py-2 border-y border-r " href="/compendium/sma?page=2">2</a><a class="px-4 py-2 border-y border-r " href="/compendium/sma?page=3">3</a><a class="px-4 py-2 border-y border-r " href="/compendium/sma?page=4">4</a><a class="px-4 py-2 border-y border-r " href="/compendium/sma?page=5">5</a><a class="px-4 py-2 border-y border-r " href="/compendium/sma?page=6">6</a><a class="px-4 py-2 border-y border-r " href="/compendium/sma?page=7">7</a><a class="px-4 py-2 border-y border-r" 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","updatedOn":null,"authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"_id":"e5cbaded-844d-4bfd-adf4-de2b8a618ee5","title":"New Research Unearths Evidence of Global Metabolic Disruption in Patients With SMA","published":"2024-09-21T14:28:00.000Z","contentCategory":{"_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2"},"factCheckAuthors":null},{"title":"Decision-Making in SMA Complicated by Insufficient Treatment Comparison","published":"2024-09-19T13:11:00.000Z","contentCategory":{"_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z"},"authors":[{"displayName":"Nameera Temkar, MD","url":"nameera-temkar-md"}],"factCheckAuthors":null,"_id":"7fe71e96-69a8-4b29-b225-d4be761f5fc6","url":"decision-making-in-sma-complicated-by-insufficient-treatment-comparison","thumbnail":{"alt":"Clinical decision-making in SMA could benefit from more comprehensive trial data comparing SMA treatments | image credit: HASAN - stock.adobe.com","caption":"Clinical decision-making in SMA could benefit from more comprehensive trial data comparing SMA treatments | image credit: HASAN - stock.adobe.com","asset":{"_ref":"image-e32d368ab659149e3f5832f8d1ee9c58ea889e90-5376x3584-jpg","_type":"reference"},"_type":"mainImage"},"summary":"Clinical decision-making can be difficult for patients with SMA due to the lack of clinical trials comparing treatment options. ","updatedOn":null},{"title":"Higher-Dose Nusinersen Meets Primary End Point in Phase 2/3 DEVOTE Study for Spinal Muscular Atrophy","url":"higher-dose-nusinersen-meets-primary-end-point-in-phase-2-3-devote-study-for-spinal-muscular-atrophy","published":"2024-09-17T12:02:00.000Z","updatedOn":null,"_id":"9ac2f771-de4b-4710-986f-6b80d2029730","summary":"Based on the positive topline data, the company plans to submit for regulatory approval for a higher dose of nusinersen for spinal muscular atrophy (SMA) treatment.","contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"authors":[{"displayName":"Isabella Ciccone, MPH","url":"isabella-ciccone-mph"}],"factCheckAuthors":[{"displayName":"Marco Meglio","url":"marco-meglio"}],"thumbnail":{"_type":"mainImage","alt":"Nusinersen was the first FDA-approved drug for the treatment of SMA in pediatric and adult patients in December 2016 | image credit: AGPhotography - stock.adobe.come","caption":"Nusinersen was the first FDA-approved drug for the treatment of SMA in pediatric and adult patients in December 2016 | image credit: AGPhotography - stock.adobe.come","asset":{"_ref":"image-567de38f26ce65e5f995fdab0a6990db7a0b9b02-3508x2480-jpg","_type":"reference"}}},{"url":"fatiguability-in-sma-may-benefit-from-nusinersen-but-further-research-is-needed","thumbnail":{"_type":"mainImage","alt":"The 6-Minute Walking Test is a common tool for measuring fatiguability in SMA | image credit: Antonioguillem - stock.adobe.com","caption":"The 6-Minute Walking Test is a common tool for measuring fatiguability in SMA | image credit: Antonioguillem - stock.adobe.com","asset":{"_ref":"image-d8de599bce63e2591c72b91f754893b502b21685-5250x3500-jpg","_type":"reference"}},"summary":"Findings from an Italian cohort suggest that nusinersen may benefit fatigue measures in patients with spinal muscular atrophy (SMA) type III; however, drawing definitive conclusions in this area remains difficult. ","published":"2024-08-28T13:16:00.000Z","updatedOn":null,"contentCategory":{"_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles"},"factCheckAuthors":null,"_id":"3fbe11dd-087b-4f7d-9815-31f8374cf540","title":"Nusinersen May Improve Fatiguability in SMA, but More Research Is Needed ","authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}]},{"url":"smn-depletion-may-increase-risk-of-liver-damage-fatty-liver-in-sma","thumbnail":{"_type":"mainImage","alt":"The SMN protein is expressed thoughout many body tissues types, including the liver | image credit: Rasi - stock.adobe.com","caption":"The SMN protein is expressed thoughout many body tissues types, including the liver | image credit: Rasi - stock.adobe.com","asset":{"_ref":"image-ea840df9cead0ba4acb662a60d766b97f74cf9d0-4200x2700-jpg","_type":"reference"}},"authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"title":"SMN Depletion May Increase Risk of Liver Damage, Fatty Liver in SMA","summary":"A retrospective, single-center cohort study utilized liver ultrasound, serum collection, and proteome analysis to draw associations between spinal motor neuron (SMN) protein depletion and heightened risk for fatty liver disease in spinal muscular atrophy (SMA). ","published":"2024-08-22T14:36:00.000Z","updatedOn":null,"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"factCheckAuthors":null,"_id":"244b292b-f84e-4894-a7bb-d764ab114c74"},{"summary":"In honor of Spinal Muscular Atrophy (SMA) Awareness Month, Abraham Homer, MS, the gaming technology supervisor and creative technologist at Children's Hospital Colorado, shared how virtual reality transformed care for patients with SMA.","published":"2024-08-21T13:23:00.000Z","_id":"e3b3a6d0-c7c5-461d-8442-6a74c3b621ff","title":"Virtual Reality Is a Game-Changer During Blood Draws for Pediatric Patients With SMA","url":"virtual-reality-is-a-game-changer-during-blood-draws-for-pediatric-patients-with-sma","thumbnail":{"asset":{"_ref":"image-59c533995a73aa1f3c94a2a54a665dfd69dab299-5477x2739-jpg","_type":"reference"},"_type":"mainImage","alt":"VR can reduce fear and anxiety associated with drawing blood in patients with SMA | image credit: matrosovv - stock.adobe.com","caption":"VR can reduce fear and anxiety associated with drawing blood in patients with SMA | image credit: matrosovv - stock.adobe.com"},"updatedOn":null,"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"authors":[{"displayName":"Abraham Homer, MS","url":"abraham-homer-ms"}],"factCheckAuthors":null},{"title":"New Motor Milestones Achieved or Sustained With Apitegromab in Nonambulatory SMA","summary":"The extension of the TOPAZ study provided support for the long-term benefits and safety of apitegromab therapy in patients with spinal muscular atrophy (SMA) at 36 months. 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","published":"2024-07-26T16:18:38.464Z","updatedOn":null,"authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"_id":"ff65669b-d2f7-4de6-9890-5d50af2351f5","title":"Potential Biomarkers Predict Disease Severity and Treatment Response in SMA","thumbnail":{"_type":"mainImage","alt":"SMA spinal muscular atrophy | Image Credit: © Rochu_2008 - stock.adobe.com","asset":{"_type":"reference","_ref":"image-c1d414e04045697dbdc36f1594c777e582892f76-1200x800-jpg"}},"contentCategory":{"_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z"},"factCheckAuthors":null},{"updatedOn":null,"authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"factCheckAuthors":null,"url":"sma-birth-prevalence-in-us-lower-than-expected-new-report-finds","published":"2024-07-19T14:30:00.000Z","thumbnail":{"_type":"mainImage","alt":"Globally, SMA is diagnosed for an estimated 1 in every 10,000 births | image credit: Alexander Raths - stock.adobe.com","caption":"Globally, SMA is diagnosed for an estimated 1 in every 10,000 births | image credit: Alexander Raths - stock.adobe.com","asset":{"_ref":"image-8b9195e98cf6fccba0be11f9f3f9e719a70dc670-6048x4032-jpg","_type":"reference"}},"summary":"A new report provided updated figures estimated the birth prevalence of spinal muscular atrophy (SMA) in the US. 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","published":"2024-07-12T16:17:00.000Z"},{"authors":[{"displayName":"Marco Meglio","url":"marco-meglio"}],"_id":"84ca85fb-1f48-4c0b-8695-39219dbb9214","title":"Final Readout of FIREFISH Study Showcases Risdiplam’s Pronounced Effects in Spinal Muscular Atrophy","thumbnail":{"_type":"mainImage","alt":"Risdiplam demonstrated great 5-year efficacy for improving motor functioning and feeding capabilities in children with type I SMA | image credit: Sittipol - stock.adobe.com","caption":"Risdiplam demonstrated great 5-year efficacy for improving motor functioning and feeding capabilities in children with type I SMA | image credit: Sittipol - stock.adobe.com","asset":{"_ref":"image-66c94cf99b709474dd3b4c3e1b26aecec7b34c92-6720x2880-jpg","_type":"reference"}},"updatedOn":null,"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"factCheckAuthors":null,"url":"final-readout-of-firefish-study-showcases-risdiplam-s-pronounced-effects-in-spinal-muscular-atrophy","summary":"The FIREFISH study showed that most children with type I spinal muscular atrophy (SMA) maintained or improved motor functions and feeding abilities over 5 years when treated with risdiplam.","published":"2024-07-03T13:52:00.000Z"},{"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"_id":"b7821164-0195-4105-889f-51c681c514e9","title":"Qualitative Study Explores Caregiver Experiences in SMA Treatment","thumbnail":{"alt":"Medical Caregiver Concept | image credit: Alexis Scholtz - stock.adobe.com","caption":"Medical Caregiver Concept | image credit: Alexis Scholtz - stock.adobe.com","asset":{"_ref":"image-86dd6d493a2e8985d74713f4e5ff7c43297f682b-7934x5292-jpg","_type":"reference"},"_type":"mainImage"},"published":"2023-12-31T15:33:00.000Z","url":"qualitative-study-explores-caregiver-experiences-in-sma-treatment","summary":"This qualitative study conducted interviews with caregivers of patients with spinal muscular atrophy (SMA) to explore their unique experiences and challenges in navigating health systems to access disease-modifying therapies. ","updatedOn":null,"factCheckAuthors":null},{"title":"More-Expensive SMA Medication Use Led to Lower Health Care Resource Utilization","summary":"Patients with spinal muscular atrophy (SMA) treated with a more expensive medication were found to have higher pharmacy costs but lower SMA-related health care resource utilization and medical costs compared with patients receiving standard-of-care nusinersen monotherapy.","published":"2023-12-27T20:10:00.000Z","updatedOn":null,"contentCategory":{"name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory"},"authors":[{"displayName":"Skylar Jeremias","url":"skylar-jeremias"}],"_id":"73735360-b40c-48c9-a539-e917a128ad70","url":"more-expensive-sma-medication-use-led-to-lower-health-care-resource-utilization","thumbnail":{"_type":"mainImage","alt":"hospital | Image credit: VILevi - stock.adobe.com","asset":{"_ref":"image-66c1591b2bef5d67825d9c88b2fdb7bf9429f3d2-4577x2647-jpg","_type":"reference"}},"factCheckAuthors":null},{"thumbnail":{"asset":{"_ref":"image-e43167f246727ae6a96a64e67a48ed31585b9839-6720x4480-jpg","_type":"reference"},"_type":"mainImage","alt":"Heavier Baby | image credit: Leka - stock.adobe.com","caption":"Heavier Baby | image credit: Leka - stock.adobe.com"},"published":"2023-12-26T12:49:00.000Z","updatedOn":null,"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"factCheckAuthors":null,"_id":"1481cdff-de96-4003-9e07-0b2f35fd0371","title":"Onasemnogene Abeparvovec Effective in Treatment of Older, Heavier Children with SMA","url":"onasemnogene-abeparvovec-effective-in-treatment-of-older-heavier-children-with-sma","summary":"A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1. "},{"title":"Alternative Method of Nusinersen Administration Identified for SMA Cases With Spinal Deformities","contentCategory":{"_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z"},"factCheckAuthors":null,"_id":"ae8f88fd-6316-4483-90f7-08c094022096","thumbnail":{"_type":"mainImage","alt":"Lumbar Region Model | image credit: Crystal light - stock.adobe.com","caption":"Lumbar Region Model | image credit: Crystal light - stock.adobe.com","asset":{"_ref":"image-c53dc9cdac5dafecb4c926067d979f0b8bb7ad44-3600x2400-jpg","_type":"reference"}},"summary":"A retrospective, single-center study out of the Czech Republic tested the safety and efficacy of unilateral interlaminar fenestration (UILF) on the convex side in cases of SMA that also exhibit severe spinal deformities. ","published":"2023-12-20T19:23:04.770Z","updatedOn":null,"authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"url":"alternative-method-of-nusinersen-administration-identified-for-sma-cases-with-spinal-deformities"},{"title":"Top 5 Most-Read Spinal Muscular Atrophy Content 2023","url":"top-5-most-read-sma-content-2023","authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"factCheckAuthors":null,"_id":"11c72011-f179-47f9-a9f3-f48e12de8d75","thumbnail":{"_type":"mainImage","alt":"Top 5 SMA Content 2023","asset":{"_ref":"image-72b4a0733f36413ab54280a85487ee24de1adb09-3187x1625-png","_type":"reference"}},"summary":"This year’s most-read spinal muscular atrophy(SMA) articles explored mental health, new technologies, caregiver well-being, and more. ","published":"2023-12-16T14:38:00.000Z","updatedOn":null},{"title":"Acetaminophen Use Requires Careful Dosing in Patients With SMA, Study Says","summary":"A non-randomized, open-label, single-site clinical trial evaluated the safety and pharmacokinetics of acetaminophen intake in adults and children with spinal muscular atrophy (SMA) type II.","published":"2023-12-06T17:52:00.000Z","factCheckAuthors":null,"_id":"dc8b3302-7f71-49fe-89b1-f6b87c1cfd24","thumbnail":{"alt":"Acetaminophen Bottle | image credit: luchschenF - stock.adobe.com","caption":"Acetaminophen Bottle | image credit: luchschenF - stock.adobe.com","asset":{"_ref":"image-fe283a2d3e21b36ebe89c1c3ae7a9636dbe6584e-2800x1868-jpg","_type":"reference"},"_type":"mainImage"},"updatedOn":null,"contentCategory":{"_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh","_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z"},"authors":[{"displayName":"Kyle Munz","url":"kyle-munz"}],"url":"acetaminophen-use-requires-careful-dosing-in-patients-with-sma-study-says"},{"thumbnail":{"caption":"Cognitive Impairment Model | image credit: Pol Solé - stock.adobe.com","asset":{"_ref":"image-624b54d83c3bcbdcd3d504ac7f1c494ba3a101a8-4608x3072-jpg","_type":"reference"},"_type":"mainImage","alt":"Cognitive Impairment Model | image credit: Pol Solé - stock.adobe.com"},"summary":"A cross-sectional study suggests that spinal muscular atrophy (SMA) can contribute to cognitive impairment. 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Lurie Children’s Hospital of Chicago and assistant professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine."}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_type":"span","marks":["underline"],"text":"Transcript:","_key":"3f660fcaaac7"}],"_type":"block","style":"normal","_key":"2083a6200c29"},{"_type":"block","style":"normal","_key":"143fcf899fd4","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Now that there are 3 treatments options for patients with spinal muscular atrophy [SMA], do you foresee any clinical guidelines being developed and if so, what do you think would be their focus?","_key":"cf33ddb4dcb6"}]},{"children":[{"_key":"b0346af2ed87","_type":"span","marks":["strong"],"text":"Rao:"},{"marks":[],"text":" I think we'd have been, over time, modifying our standard of care guidelines. The most recent modification was 2 to 3 years ago and that happened after a long time after the previous modification. This modification to the standard of care guidelines was pushed because nusinersen [Spinraza] was approved. Now, we have 2 drugs [approved] after that. So, I'm confident that we need more modification or change in our standard of care, especially because our natural history, in terms of what an individual with SMA looks like, is absolutely modified. It's different. And thank goodness, it's different. What do we do with these modified natural histories is going to be the subject of the new standard of care guidelines. The child that was not destined to sit up or walk is now sitting up and walking. What does that mean for bone health? What does that mean for their orthopedic manipulations in the future? How do we deal with scoliosis in children who were not supposed to get scoliosis? And vice versa? It's a very vast question and I don't think we'll be able to deal with every aspect of it today, but I think we will need to change and modify our standard of care guidelines. And those standard of care guidelines will also need to talk about or touch upon what we think about combination therapies.\nMy analogy to that I use comes from the world of oncology. When we started looking at cancers in children, we started using drugs and we found that those drugs were riddled with side effects, but they had some benefit of prolonging life. So, we kept using them. Then, we found out that we could make better drugs but we realized that we actually had to combine those drugs. Over time, we realized we not only had to combine them, but we also had to look at the genetics of the particular cancer to personalize those drugs. I think we're moving in that same direction with SMA. We started with a drug, and there were nowhere close to the side effects that we experienced with the cancer drugs, so that's definitely better. The cost of the drugs is sometimes limiting in terms of how the insurance companies allow us to combine drugs. We have to look at each child and say what stage they are, what developmental milestones they haven't hit. What is the gap that we still have in terms of these children attaining the next level? What standard of care guidelines should be put into place, as well as combination therapies and potentially new therapies in the future? So, it's a very it's a very hopeful world, but it's a world and a future filled with a ton of questions that we need to answer before we get there.","_key":"7b16a7fb1f1e","_type":"span"}],"_type":"block","style":"normal","_key":"ffe53a7cf134","markDefs":[]}],"title":"Dr Vamshi Rao Discusses Possible Future Guidelines for SMA Care","summary":"Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. 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Lurie Children’s Hospital of Chicago and assistant professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine.","_key":"1d3d6f5024c4","_type":"span","marks":["em"]}]},{"markDefs":[],"children":[{"_type":"span","marks":["underline"],"text":"Transcript:","_key":"a1c091fcc0150"}],"_type":"block","style":"normal","_key":"9bf8c9198737"},{"children":[{"text":"What future do you see for combination therapies in SMA and how will they affect patient outcomes?","_key":"e8930b2660180","_type":"span","marks":["strong"]}],"_type":"block","style":"normal","_key":"b564a6c31b80","markDefs":[]},{"children":[{"marks":["strong"],"text":"Rao: ","_key":"f6807c51c74e0","_type":"span"},{"_type":"span","marks":[],"text":"I think we are definitely seeing a push for combination therapies from the patient point of view but also from the scientific community in general. I think what we are struggling with, of course, is lack of data in terms of using combination therapies in the real world. We have 3 drugs approved. You would think that we would have the discretion of prescribing those drugs as a combination, but it's hard because there is no clinical trial evidence of that. So, I think most of the sponsors and pharmaceutical companies that own these drugs are looking forward to some data out there that can result in a combination product, whether it is a trial where their drug is the first to be given or whether their drug is the one that follows a drug. For example, because nusinersen [Spinraza] was approved in 2016 and most folks started with nusinersen, there are now trials looking at a whether coming in with a drug after nusinersen, such as, Zogensma [onasemnogene abeparvovec] or Evrysdi [risdiplam], would be more beneficial. More so, I think people are also looking at safety of when to use combination therapy.","_key":"f6807c51c74e1"}],"_type":"block","style":"normal","_key":"9af7a0475035","markDefs":[]},{"style":"normal","_key":"97aa138431b9","markDefs":[],"children":[{"_key":"7a6b2ffdaab50","_type":"span","marks":[],"text":"Right now, there isn't much data out there, [except for] some publications, about real world experiences. A group that I work with published a paper where we looked at children who had nusinersen or Zolgensma and then got the other drug, whether it was nusinersen or Zolgensma. So, it's small studies. I don't think we can generalize results yet. Definitely, I do see that combination therapies are going to be the wave of the future and I say that because unless you have 1 drug that has been instituted early in life, that leads to a child or an individual who looks like a non-SMA child, there is going to be a quest for something better, something more. So, if you have had a drug that makes you attain motor milestones, and jumpstarts your development, but you don't reach the curve or follow the curve of development of a non-SMA trial, then there's still some scope to bridge the gap. That's where the question about what's next, whether it's a new drug or a combination of drugs, is going to happen."}],"_type":"block"}],"title":"Dr Vamshi Rao Discusses the Future of Combination Therapy in SMA","internalTag":["combination therapy","sma","spinal muscular atrophy","spinraza","zolgensma","evrysdi"],"seoTag":["combination therapy","sma","spinal muscular atrophy","spinraza","zolgensma","evrysdi"],"authorMapping":null},{"is_visible":true,"title":"Dr Vamshi Rao Discusses Safety, Efficacy of SMA Treatments","_type":"article","url":{"current":"dr-vamshi-rao-discusses-safety-efficacy-of-sma-treatments","_type":"slug"},"source":null,"_rev":"Mh7rwKGTOa5fWvi8Dytw2e","body":[{"children":[{"_type":"span","marks":["em"],"text":"","_key":"252e8beba851"}],"_type":"block","style":"normal","_key":"2d3ddb6a88d9","markDefs":[]},{"_type":"video","videoID":"6230144479001","source":"brightcove","_key":"b4e322f62a78"},{"markDefs":[],"children":[{"_type":"span","marks":["em"],"text":"Vamshi Rao, MD, is an attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago and assistant professor of Pediatrics (Neurology and Epilepsy) at the Northwestern University Feinberg School of Medicine.","_key":"ef696af27450"}],"_type":"block","style":"normal","_key":"53a3719c2b7f"},{"_key":"8f92a1944e28","markDefs":[],"children":[{"_type":"span","marks":["underline"],"text":"Transcript","_key":"e9d2e3d2cd980"},{"_type":"span","marks":[],"text":":","_key":"e9d2e3d2cd981"}],"_type":"block","style":"normal"},{"_key":"d570c0baced7","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Are there any complications that can result from any of the spinal muscular atrophy (SMA) therapies that patients or caregivers should be aware of prior to starting?","_key":"66e21654ed3b0"}],"_type":"block","style":"normal"},{"children":[{"_key":"eb656026a9e6","_type":"span","marks":["strong"],"text":"Rao: "},{"_key":"fecd5f805b6f0","_type":"span","marks":[],"text":"Safety was looked at in a very astute observation sense with all the clinical trials. I have to say that different medications have different safety markers. So, with a medication like nusinersen (Spinraza), the practical pointers with safety included safety that was related to the drug and risks that had to be looked at with the lumbar puncture that was used to give the product."}],"_type":"block","style":"normal","_key":"9d62b53a390b","markDefs":[]},{"_key":"d3ada4b38fdb","markDefs":[],"children":[{"_type":"span","marks":[],"text":"As far as the drug itself was concerned, in the clinical trials, it was shown that there was a slight risk of proteinuria, or protein in the urine, which was related to a synthetic drug that was going through the body system and had to be filtered through the kidney. So, the FDA had asked us to look at urine analysis. I have to say that in, in my real world experience, at least in pediatrics, which is what I deal with, I did not see any safety signals that were significant enough for us to warrant further analysis after taking urine. We did not see any children at our institution with any signs of renal failure.","_key":"b055fc4152140"}],"_type":"block","style":"normal"},{"style":"normal","_key":"d84eaad686af","markDefs":[],"children":[{"_type":"span","marks":[],"text":"The other safety factors that were taken into consideration was because you were doing a procedure such as lumbar puncture, the FDA had asked us to look at platelet count and coagulation, for preoperative safety to make sure that we weren't dealing with anybody with coagulation abnormalities and then doing a lumbar puncture. And I have to say that over time, the community as such has rallied to actually not have that done because most of us don't feel that we do it in the real world anyway, let alone with Spinraza. So, we have been trying to be very judicious, especially balancing the need to draw blood from a child as opposed to the benefits of it.","_key":"efd868b6689e0"}],"_type":"block"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"Other than that, there have been safety signals that have been very rare, I would say. Constipation has been one of the signals. But, in the real world, we have found that it's a very, very safe drug and we haven't had any complications. I think we definitely are very watchful about complications that can arise from getting a lumbar puncture. And one of the biggest ones, and the most common ones, that we see is people who can get a postlumbar puncture headache. So, we guard against that by making sure the child is well hydrated or if the child needs hydration after the procedure. We've definitely had some instances of that. As an institution, we have used a different kind of needle to do a lumbar puncture; it's called an atraumatic needle. In literature, it has been shown and we have seen that it decreases the incidence of post lumbar puncture headaches.","_key":"0522d7ddbd0f0"}],"_type":"block","style":"normal","_key":"4755d122a17c"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"Now, safety as a signal from the onasemnogene abeparvovec (Zolgensma) trial, which is loosely referred to as the gene therapy drug, is definitely something that requires more observation because there is an elevation of liver enzymes after the drug is administered and a possible fall in platelets within the first week after the drug is administered. So, it being a medication that is enveloped in a virus, there's definitely an immunogenic reaction that has more to do with the virus trying to be eliminated from the body by the liver. One thing that has been a guideline to guard against overt elevation is the use of steroids prior to the infusion and the use of steroids during the first month and a very close observation of the liver enzymes and the platelets. So, these children get blood draws pretty much from every week to every 2 weeks to watch the liver enzyme levels and platelets, in addition to another signal that was observed in the clinical trial, which is the triponin I.","_key":"3d4d6f8b73d30"}],"_type":"block","style":"normal","_key":"c26c5adc9603"},{"markDefs":[],"children":[{"text":"So, in the real world, I would say that we definitely keep a very close eye on the liver enzymes. We have seen elevations of liver enzymes where we had to prolong the use of steroids and sometimes increase the dose of steroids to combat the liver enzymes. But in our institution, at our sort of practice area, we haven't had any cases of liver failure, or any cases of liver damage but we have had cases where we've had to use the steroids for longer than what we anticipated. All those children are doing well. So, definitely something to watch for.","_key":"684c6b0f00480","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"0795dba5d3a8"},{"children":[{"text":"I haven't really talked about the third drug that has been approved because it got recently approved, which is risdiplam (Evrysdi). 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I can speak for the state of Illinois because that is where I practice. Newborn screening was introduced as a pilot somewhere between May and July 2020 and rapidly took over as a permanent program after that. All of the children that are being born in the state of Illinois are definitely going through newborn screening and COVID-19 has not affected that. As far as access to treatment, access to treatment at a particular institution for a therapy that requires children to come to the hospital, such as Spinraza [nusinersen] that is given through lumbar puncture, or on a *","_key":"3e6420a63f4f0"},{"_type":"span","marks":["strong"],"text":"17 hour power glycosyltransferase*","_key":"3e6420a63f4f1"},{"text":" that is given through IV [intravenous injection] requires children to come to the hospital.","_key":"3e6420a63f4f2","_type":"span","marks":[]}],"_type":"block"},{"markDefs":[],"children":[{"marks":[],"text":"Guidelines that required COVID-19 precautions were in place in most people places, and guidelines have to be practiced. But at the same time, a very risk-benefit analysis was done in terms of exposure vs children getting treatment and I'm happy to say that, at least at our institution, as we really looked at these guidelines [with] a committee that was formed to assess whether children were going to come in for their regular treatments, and clinical trials, it was found that the risk of not getting the medication vs the risk of getting COVID was definitely in favor of decline with the underlying estimate if they did not get their medication. So all these children were allowed to have their disease modifying therapies given to them at our hospital.","_key":"3e422618643e0","_type":"span"}],"_type":"block","style":"normal","_key":"fb06a1011e9a"},{"_key":"38614a60237f","markDefs":[],"children":[{"_type":"span","marks":[],"text":"I definitely think that access was affected at some institutions and I do think access was affected around the country, in places where a lot of travel was involved in whether kids were coming from rural communities, or coming from very far off, mountainous regions to their nearest center that was giving them these drugs. 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Lurie Children’s Hospital of Chicago and assistant professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine.","_key":"3bbb8eb9ecf8","_type":"span","marks":["em"]}],"_type":"block","style":"normal","_key":"1b2f74a5adf9"},{"markDefs":[],"children":[{"marks":["strong"],"text":"What does a “good prognosis” look like in SMA, now that there is a disease- modifying therapy?","_key":"528744fc227d0","_type":"span"}],"_type":"block","style":"normal","_key":"5be8a220331e"},{"_key":"5c237387e9bc","markDefs":[],"children":[{"marks":["strong"],"text":"Rao: ","_key":"1fd570157ed40","_type":"span"},{"_type":"span","marks":[],"text":"The prognosis is definitely better than what we anticipated when the disease-modifying therapies were not out there. It still depends on the type of SMA that we're looking at. There are 3 types of SMA and the one that is most severe is the type 1, where kids used to not survive beyond the age of 18 months and they never achieved the ability to even sit up.","_key":"1fd570157ed41"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"I think the greatest difference these drugs have made is in type 1 SMA because the endpoints of the clinical trials were either death or permanent ventilation. If you look at that as a prognosis, we are definitely way beyond that. We don't have deaths occurring because of SMA type 1 and as far as the need for permanent ventilation, that has also decreased with a very important caveat, which is, when do you treat these children with the disease-modifying therapies. As we found out in the clinical trials, the earlier the treatment, the better the prognosis. So, the pivotal clinical trials for SMA type 1 showed that these kids [children treated under 6 months of age] not only stabilized but also achieved more motor milestones, which they had never done before.","_key":"98d0d48c37b00"}],"_type":"block","style":"normal","_key":"15d694127918"},{"children":[{"_type":"span","marks":[],"text":"As far as treating them even prior to when we think is the time that the motor neurons die, somewhere around 4 to 6 weeks of age, these kids do very well. In fact, we have some children that have been treated after the drugs were approved, the real world or commercial data, where the children actually don't look much different from children without SMA. So, that is a very, very phenomenal prognostic benchmark for us—these kids are sitting up, some of them are crawling, and there are a few kids that are walking. And these are kids that were never supposed to be alive after 18 months of age.","_key":"9e54bf1c8d460"}],"_type":"block","style":"normal","_key":"0e319700b528","markDefs":[]},{"_type":"block","style":"normal","_key":"73576c25f77d","markDefs":[],"children":[{"_type":"span","marks":[],"text":"So, to answer your question, in terms of prognosis, it's very different for type 1, type 2, and type 3. Kids with SMA type 2 were kids who were going to sit up but would never achieve the ability to walk. Kids, young adults, and adults with type 3 were individuals who would walk but could become nonambulatory over time. There's still a lot of clinical trials that are being done to assess that and a lot of data that is coming out from the real world as to what exactly the prognosis is going to look like for type 2 and type 3. But it does look like that there's an improvement. 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severity spinal muscular atrophy (SMA).","_key":"3b827a7a2e63","_type":"span"}]},{"markDefs":[],"children":[{"_type":"span","marks":["underline","strong"],"text":"Transcript","_key":"9ff7c42614c9"}],"_type":"block","style":"normal","_key":"fec5b6e87248"},{"_type":"block","_key":"dc30e05f9a26","markDefs":[],"children":[{"text":"What are your thoughts regarding whether gender has any effects on disease severity? 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SMA.","_key":"3b827a7a2e63"}],"_type":"block","_key":"3d49b8dd040f"},{"style":"normal","_key":"fec5b6e87248","markDefs":[],"children":[{"_type":"span","marks":["underline","strong"],"text":"Transcript","_key":"9ff7c42614c9"}],"_type":"block"},{"style":"normal","_key":"dc30e05f9a26","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"What do we know about the importance of treating early in infants with SMA?","_key":"588efd925620"}],"_type":"block"},{"_key":"53f2a940044f","markDefs":[],"children":[{"marks":["strong"],"text":"Jarecki: ","_key":"1cddac0fc1f2","_type":"span"},{"_key":"947eab9d9ed1","_type":"span","marks":[],"text":"So, SMA is a motor neuron disease. Motor neurons are dysfunctional and eventually lost. Neurons are a cell type that cannot be regenerated once lost. So, we know that it is important to intervene prior to the loss of motor neurons. And for many years, we've known that mouse models of SMA require an early window of treatment for SMN- [survivor motor neuron] enhancing drugs to have full benefit. And in patients, we've known for many years that electrophysiological experiments in SMA Type 1 infants have denervation of muscle approaching 90% by 6 months of age."}],"_type":"block"},{"_key":"bc720e3c4edc","markDefs":[],"children":[{"marks":[],"text":"So, we believe it's very important that infants are treated presymptomatically through newborn screening. And there's been further clinical trial data and there are clinical trials looking at presymptomatic infants with all of the FDA approved drugs, but the only one that's been peer reviewed and published so far was called the NURTURE trial from Biogen looking at Spinraza [nusinersen].","_key":"e27f819188ec","_type":"span"}],"_type":"block"},{"markDefs":[],"children":[{"text":"There, they've shown dramatic benefits over symptomatic treatment. With presymptomatic treatment of infants under 6 months of age, you now have a Type 1 infant who would not sit, typically not be able to eat on their own, and need extensive respiratory support. Now, 100% of those babies treated presymptomatically are sitting, 100% are crawling, 100% are standing, and 88% are walking independently.","_key":"dae89f7e08de","_type":"span","marks":[]}],"_type":"block","_key":"a5eb4b0a7dbf"},{"_type":"block","_key":"d0cb4e41d06d","markDefs":[],"children":[{"_type":"span","marks":[],"text":"So, there really are dramatic gains with presymptomatic treatment was Spinraza. And we expect to see the same with the other SMN upregulating drugs when those results are published.","_key":"4a4753f694c0"}]}],"url":{"current":"dr-jill-jarecki-discusses-the-early-sma-treatment-for-infants","_type":"slug"},"_rev":"bfpFLB9Rnq5SkaWyzOsLwT","link":"/view/dr-jill-jarecki-discusses-the-early-sma-treatment-for-infants","updatedOn":null,"source":null,"title":"Dr Jill Jarecki Discusses the Early SMA Treatment for Infants","_createdAt":"2020-10-13T17:01:08Z","is_visible":true,"_updatedAt":"2020-10-13T18:47:58Z","internalTag":["spinal muscular atrophy"],"published":"2020-10-25T11:00:00.000Z","contentCategory":{"_type":"contentCategory","name":"Videos","_id":"ee14ccb3-3542-4414-9046-927be1198c76","_updatedAt":"2020-04-03T20:03:44Z","_createdAt":"2020-04-03T20:03:44Z","_rev":"Yw6MEKZDMdk6hC2JCPjfiB"},"_type":"article","_id":"14ac5cb0-39f6-4af2-9834-999e68fb2aef","seoTag":["spinal muscular atrophy"],"summary":"Early treatment of infants with spinal muscular atrophy (SMA) can 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SMA.","_key":"3b827a7a2e63"}]},{"style":"normal","_key":"fec5b6e87248","markDefs":[],"children":[{"text":"Transcript","_key":"9ff7c42614c9","_type":"span","marks":["underline","strong"]}],"_type":"block"},{"_type":"block","style":"normal","_key":"dc30e05f9a26","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"What do treatment or management options look like for people with milder subtypes of SMA?","_key":"d5f40846e47b"}]},{"_key":"5e3f74ad217e","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Schroth: ","_key":"026f62eeb839"},{"marks":[],"text":"Treatment and management options for people with milder subtypes of SMA really are the same. SMA's a progressive neurodegenerative disease, meaning that any person living with SMA will experience progressive loss of function over time. SMA is caused by deletion or mutation of the survivor motor neuron gene 1 [SMN1].","_key":"d7eb2fa75b0a","_type":"span"}],"_type":"block"},{"children":[{"marks":[],"text":"In a healthy person, this gene produces a protein that is critical to the function of nerves that control our muscles. Without it, those nerves cannot properly function and eventually die leading to debilitating and often fatal weakness of the muscles. When that happens is determined by the number of backup genes known as SMN2 genes a person has. So, it is not a matter of if symptoms start or progress, but rather when they do.","_key":"1454dd3bfd57","_type":"span"}],"_type":"block","_key":"b33aef46bbd7","markDefs":[]},{"markDefs":[],"children":[{"_key":"3f3f7a7f90ee","_type":"span","marks":[],"text":"There are treatments approved for all types of SMA and 2 of these medications have age eligibility criteria, however, the available treatments are the same regardless of motor strength."}],"_type":"block","_key":"7ed817740cc9"},{"_type":"block","_key":"9c1292be0815","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Jarecki: ","_key":"93325ec8dec3"},{"_type":"span","marks":[],"text":"Just to elaborate a little bit on what Dr. Schroth said, we have a traditionally 4 types of SMA. We have Type 1 SMA, which is the most severe form of SMA, which typically correlates to 2 copies of SMN2, the backup gene she mentioned. Type 2 SMA, which typically correlates to 3 copies of SMN2. And then, Type 3 SMA is where you have a 50/50 split of patients with 3 and 4 copies of SMN2. And then Type 4, which is a very rare adult onset of SMA where you could have even more than 4 copies of SMN2.","_key":"1e6992f5a1d1"}]},{"markDefs":[],"children":[{"text":"Each of these types traditionally is defined by maximal motor function. So, Type 1 children would be considered nonsitters. Type 2 children would be sitters. Type 3 patients would walk, but lose the ability to walk. And then, Type 4 patients would be the mildest form of the disease. But we're excited because those labels are becoming obsolete with the ability of these SMN upregulating approved drugs to improve the phenotype.","_key":"a4f8ed2cf54e","_type":"span","marks":[]}],"_type":"block","_key":"086c9119150d"},{"markDefs":[],"children":[{"marks":[],"text":"For example, in our patient reported database, we now have 37% of people who self-identify, having Type 1 SMA being able to sit. 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Schroth","asset":{"_ref":"image-2fd2d97d94e19aea8359b7ea9e52763a2285768c-1280x720-png","_type":"reference"}},"_type":"youtube","_key":"ceea7b527299","url":"https://youtu.be/ZdyWUELj8UQ"},{"_key":"206ad2b618e7","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"The latest FDA approved treatment is Evrysdi, which is an oral at-home therapy. What are the benefits of having this additional therapy available?","_key":"68fee851a51f"}],"_type":"block"},{"children":[{"_key":"864d8369daeb0","_type":"span","marks":[],"text":"The FDA approval of Evrydi (risdiplam) plan provides another treatment option for people living with SMA. The ability to take a medication at home and by mouth or feeding tube will allow more people access to an SMA treatment. With multiple treatments for us to may available, patients should work in consultation with their health care provider team to determine which treatment option is best for their specific needs."}],"_type":"block","style":"normal","_key":"cf5b662a6c0b","markDefs":[]},{"markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"How has the COVID-19 pandemic affected people with SMA and their caregivers? For instance, are they more at risk of getting infected? Do we know what impact the virus has if they do get COVID-19?","_key":"a5d65e7ac7fc0"}],"_type":"block","style":"normal","_key":"71671ad21d97"},{"style":"normal","_key":"4c99f2ca10e0","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Per feedback from our community about COVID-19, we learned that COVID-19 temporarily interfered with patients being able to receive treatments as scheduled. Monitory ","_key":"0858e881d4c50"},{"_type":"span","marks":[],"text":"care transitioned to more telemedicine delivery and providing care in-person when necessary. Similarly, clinical trial visits have been modified to conduct in-person visits when essential, and other visits were modified to be conducted virtually. ","_key":"0858e881d4c52"}],"_type":"block"},{"_type":"block","style":"normal","_key":"66c1a97f2c12","markDefs":[],"children":[{"_type":"span","marks":[],"text":"We have limited reports of people living with us may have actually having COVID-19. In general, people living with SMA avoid others who are sick. And during the winter viral season, they already were avoiding public contact as much as possible. There's not enough information available to make any conclusions about the impact of infection on those with SMA. 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