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Dystrophy</h1><div class="flex justify-center px-0 -mt-1 sm:-mt-2 align-center"></div></div><div class="w-full mb-2 border border-secondary"></div><div id="description-summary" class="text-sm text-gray-500"><div class="blockText_blockContent__TbCXh"></div><div class="clear-both"></div></div><div class="border-b-[2px] border-[#EEE] "><div class="m-2"><div class="hidden xs:flex justify-between items-center"><h2 class="text-3xl">Latest News</h2></div><div class="xs:my-6 my-4 xs:block hidden"><div class="flex"><div class="flex sm:pr-[35px] pr-[20px] flex-col w-[40%] border-r-[2px] border-[#EEE]"><div class="lg:h-[220px] h-[170px] relative bg-default-logo-background "><a class="0 w-full" href="/view/wve-n531-shows-functional-benefits-muscle-damage-reversal-in-dmd"><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-contain" 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%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.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">March 27th 2025</span><div class="py-3"><a class="lg:text-[20px] text-[19px] font-[500]" href="/view/wve-n531-shows-functional-benefits-muscle-damage-reversal-in-dmd">WVE-N531 Shows Functional Benefits, Muscle Damage Reversal in DMD</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/rose-mcnulty"><i>Rose McNulty</i></a></span></div></div><a href="/view/wve-n531-shows-functional-benefits-muscle-damage-reversal-in-dmd"><a href="/view/wve-n531-shows-functional-benefits-muscle-damage-reversal-in-dmd"><p class="mt-1 text-gray-800 text-[13px] line-clamp-6 text-hidden">WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients with Duchenne muscular dystrophy (DMD) in the phase 2 FORWARD-53 trial.</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/real-world-study-suggests-dmd-gene-therapy-is-safe-effective"><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="" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="fill" class="lg:object-cover object-contain " 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%2Fd2c61cb6916c5df55b1355585d6bf4a6bf23fbce-6720x4480.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fd2c61cb6916c5df55b1355585d6bf4a6bf23fbce-6720x4480.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fd2c61cb6916c5df55b1355585d6bf4a6bf23fbce-6720x4480.jpg%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%2Fd2c61cb6916c5df55b1355585d6bf4a6bf23fbce-6720x4480.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/real-world-study-suggests-dmd-gene-therapy-is-safe-effective"><span class="text-sm text-gray-500">March 26th 2025</span></a><div><a class="text-[14px]" href="/view/real-world-study-suggests-dmd-gene-therapy-is-safe-effective"><p class="line-clamp-4 text-hidden font-[500]">Real-World Study Suggests DMD Gene Therapy Is Safe, Effective</p></a></div><div class="py-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/rose-mcnulty"><i>Rose McNulty</i></a></span></div></div></div></a><a class="flex w-[50%] flex-col sm:pl-[35px] pl-[20px] pb-[35px] " href="/view/delandistrogene-moxeparvovec-shows-long-term-impacts-at-3-and-5-years-in-dmd"><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="" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="fill" class="lg:object-cover object-contain " 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" 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class="jsx-1215160397 w-full h-full flex flex-col overflow-hidden relative bg-white text-center"><div class="jsx-1215160397 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%2F453ff95d3edc29442e2af71658751e2f26adc78e-2500x2500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, 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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F453ff95d3edc29442e2af71658751e2f26adc78e-2500x2500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F453ff95d3edc29442e2af71658751e2f26adc78e-2500x2500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F453ff95d3edc29442e2af71658751e2f26adc78e-2500x2500.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-cover" loading="lazy"/></noscript></span></div><div class="jsx-1215160397 content-wrap"><span class="jsx-1215160397 py-3 text-sm text-gray-500 text-left"><i class="jsx-1215160397">July 18th 2024</i></span><div class="jsx-1215160397 w-full h-fit mb-4"><p class="jsx-1215160397 text-[18px] font-bold text-[#000000] text-left line-clamp-3 text-hidden">Insurance Insights: Dr Jason Shafrin Estimates DMD Insurance Value</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/sgt-003-gene-therapy-shows-potential-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/32f4d0a5f3124487aa79aa2cf6ac53ec54744a29-6000x4000.jpg?fit=crop&auto=format" alt="In the INSPIRE DUCHENNE trial, interim 90-day biopsy data showed an average microdystrophin expression of 110% among the first 3 participants in the study. | Image credit: RFBSIP - 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">February 26th 2025</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/sgt-003-gene-therapy-shows-potential-in-dmd">SGT-003 Gene Therapy Shows Potential in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/sgt-003-gene-therapy-shows-potential-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants with Duchenne muscular dystrophy (DMD) at 90 days post 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 class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/adults-with-dmd-more-than-twice-as-likely-to-experience-fractures"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/06061deade20b7548bf1379ea5954d86c9277e14-1200x675.jpg?fit=crop&auto=format" alt="Vertebral fractures are common in glucocorticoid-associated osteoporosis and are a clinical indicator of osteoporosis in young patients. | Image credit: Hugnaka - 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">February 14th 2025</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/adults-with-dmd-more-than-twice-as-likely-to-experience-fractures">Adults With DMD More Than Twice as Likely to Experience Fractures</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/adults-with-dmd-more-than-twice-as-likely-to-experience-fractures"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of osteoporosis in adult men with Duchenne muscular dystrophy (DMD). </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/ifetroban-improves-heart-function-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/00882496c7fe122f1e99a913247b7f18619fde44-1200x800.jpg?fit=crop&auto=format" alt="Ifetroban may improve LVEF in patients with DMD. - REDPIXEL - 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">February 7th 2025</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/ifetroban-improves-heart-function-in-dmd">Ifetroban Improves Heart Function in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/ifetroban-improves-heart-function-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results suggest ifetroban may improve left ventricular ejection fraction in patients with DMD. </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/embark-2-delandistrogene-moxeparvovec-shows-sustained-benefits-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/256eb619de1c0a8ceade136b979b183b7e6d80d2-1000x667.jpg?fit=crop&auto=format" alt="Duchenne muscular dystrophy | Image credit: hafakot - 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">January 28th 2025</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/embark-2-delandistrogene-moxeparvovec-shows-sustained-benefits-in-dmd">EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/embark-2-delandistrogene-moxeparvovec-shows-sustained-benefits-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD). </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/prophylactic-cardiac-medication-may-prolong-survival-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/37d115516d72b70fdccd564ea127cdbc3a31869e-1200x673.jpg?fit=crop&auto=format" alt="Heart and stethoscope | Image credit: inthasone - 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">January 24th 2025</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/prophylactic-cardiac-medication-may-prolong-survival-in-dmd">Prophylactic Cardiac Medication May Prolong Survival in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/prophylactic-cardiac-medication-may-prolong-survival-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals received such treatment, a recent study found. </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/quantifying-the-altruism-value-for-a-rare-pediatric-disease-duchenne-muscular-dystrophy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/5421358c8f117ee86126333e135f08cf1e61ded6-778x382.png?fit=crop&auto=format" alt="AJMC" 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">January 14th 2025</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/quantifying-the-altruism-value-for-a-rare-pediatric-disease-duchenne-muscular-dystrophy">Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jason-shafrin,-phd">Jason Shafrin, PhD</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/suhail-thahir-ms">Suhail Thahir, MS</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/alexa-c-klimchak-ma">Alexa C. Klimchak, MA</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ivana-audhya-msc">Ivana Audhya, MSc</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/lauren-e-sedita-ms">Lauren E. Sedita, MS</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/john-a-romley-phd">John A. Romley, PhD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/quantifying-the-altruism-value-for-a-rare-pediatric-disease-duchenne-muscular-dystrophy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 per year.</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/later-loss-of-ambulation-linked-with-better-heart-health-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/674b4a7f2c7cd38a9d9029d5d6430eaccac96d0d-5150x3648.jpg?fit=crop&auto=format" alt="Past studies have explored the association between motor and cardiac function in younger patients with DMD, but there is a lack of research on this relationship as patients with DMD age. | Image credit: Nana_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">January 13th 2025</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/later-loss-of-ambulation-linked-with-better-heart-health-in-dmd">Later Loss of Ambulation Linked With Better Heart Health in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/later-loss-of-ambulation-linked-with-better-heart-health-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with Duchenne muscular dystrophy (DMD).</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-highlights-trends-emerging-innovations-in-dmd-research"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/860ecc80e940703fd332d679608fdc354c4efb2b-6016x4016.jpg?fit=crop&auto=format" alt="lab test tubes | Image credit: kwanchaift – 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 22nd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/study-highlights-trends-emerging-innovations-in-dmd-research">Study Highlights Trends, Emerging Innovations in DMD 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/hayden-e-klein">Hayden E. Klein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/study-highlights-trends-emerging-innovations-in-dmd-research"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">An analysis revealed global trends and emerging focus areas in Duchenne muscular dystrophy (DMD) research, emphasizing the growing impact of precision medicine and gene 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 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/mri-biomarker-may-signal-early-heart-damage-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/0701c16da7f575b3d0809c6fa104ce4fc03578d6-6000x4000.jpg?fit=crop&auto=format" alt="Heart anatomy on paper | Image credit: larisikstefania – 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 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/mri-biomarker-may-signal-early-heart-damage-in-dmd">MRI Biomarker May Signal Early Heart Damage in DMD</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/mri-biomarker-may-signal-early-heart-damage-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Septal circumferential strain (Ecc) was significantly decreased in LGE-negative boys with Duchenne muscular dystrophy (DMD) and correlated to ventricle changes.</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/fda-pauses-pepgen-s-dmd-drug-trial-in-the-us"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/e9f3330b21f13fe51a104793dc091dacc5848457-5979x3923.jpg?fit=crop&auto=format" alt="Gene therapy | Image credit: Treecha – 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 19th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/fda-pauses-pepgen-s-dmd-drug-trial-in-the-us">FDA Pauses PepGen’s DMD Drug Trial in the US</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/fda-pauses-pepgen-s-dmd-drug-trial-in-the-us"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The CONNECT2-EDO51 phase 2 trial looks at PGN-EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.</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/top-5-duchenne-muscular-dystrophy-content-of-2024"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/e67fe3af32cb5a8ada28f3f9964afe9005c1ed87-1020x520.png?fit=crop&auto=format" alt="The top 5 DMD content of 2024" 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 19th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/top-5-duchenne-muscular-dystrophy-content-of-2024">Top 5 Duchenne Muscular Dystrophy Content of 2024</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/top-5-duchenne-muscular-dystrophy-content-of-2024"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">From imaging innovations to new treatment approvals, 2024 brought advancements in Duchenne muscular dystrophy care and 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/real-world-challenges-in-dmd-pmo-treatment"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d2c61cb6916c5df55b1355585d6bf4a6bf23fbce-6720x4480.jpg?fit=crop&auto=format" alt="Gene therapy graphic with DNA strands | Image credit: natali_mis – 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 25th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/real-world-challenges-in-dmd-pmo-treatment">Real-World Challenges in DMD PMO 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/hayden-e-klein">Hayden E. Klein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/real-world-challenges-in-dmd-pmo-treatment"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A claims-based study revealed high adherence to phosphorodiamidate morpholino oligomer (PMO) treatments for Duchenne muscular dystrophy (DMD), but highlights limitations in interpreting real-world effectiveness from claims data.</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/viltolarsen-slows-dmd-progression-in-small-study"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/7e20ff65b7abea493e84dace957fb80991ce7bb1-6048x4024.jpg?fit=crop&auto=format" alt="Boy with DMD using walker | Image credit: Svitlana – 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 23rd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/viltolarsen-slows-dmd-progression-in-small-study">Viltolarsen Slows DMD Progression in Small Study</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/viltolarsen-slows-dmd-progression-in-small-study"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Further research is needed to determine how once-weekly viltolarsen can slow disease progression and preserve motor function in boys and young men with Duchenne muscular dystrophy (DMD).</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/fda-accepts-resubmitted-nda-for-ataluren-in-nonsense-duchenne-muscular-dystrophy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/ebfb40d061db8cb94776018fe5f4c50f7871539b-5293x3529.jpg?fit=crop&auto=format" alt="FDA website | Image Credit: © Postmodern 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">November 22nd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/fda-accepts-resubmitted-nda-for-ataluren-in-nonsense-duchenne-muscular-dystrophy">FDA Accepts Resubmitted NDA for Ataluren in Nonsense Duchenne Muscular Dystrophy</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/fda-accepts-resubmitted-nda-for-ataluren-in-nonsense-duchenne-muscular-dystrophy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The new drug application (NDA) includes data from a global placebo-controlled, 72-week study as well as findings from the STRIDE registry, an ongoing, observational, real-world study of ataluren in routine care.</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/sarepta-halts-development-testing-of-dmd-therapy-srp-5051"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/a7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg?fit=crop&auto=format" alt="Researcher working with microplate in the laboratory | Image credit: angellodeco – 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 14th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/sarepta-halts-development-testing-of-dmd-therapy-srp-5051">Sarepta Halts Development, Testing of DMD Therapy SRP-5051</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/sarepta-halts-development-testing-of-dmd-therapy-srp-5051"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with the developer citing safety concerns, FDA feedback, and an evolving therapeutic landscape.</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/a-year-of-dmd-gene-therapy-trial-failures"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/f17e24452d6c5da3f5c3add1df744b5a59621ffe-6000x4000.jpg?fit=crop&auto=format" alt="Gene therapy research | Image credit: RFBSIP – 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: October 29th 2024 | Updated: October 30th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/a-year-of-dmd-gene-therapy-trial-failures">A Year of DMD Gene Therapy Trial Failures</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/a-year-of-dmd-gene-therapy-trial-failures"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The outcomes of the EMBARK and CIFFREO trials in boys aged 4 to 7 have raised concerns in the Duchenne muscular dystrophy (DMD) community and prompted layoffs at Pfizer.</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/studies-highlight-heavy-burden-on-caregivers-of-patients-with-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/7b01dbf92510da0d1d91407c2370108cc29fa2ac-5472x3648.jpg?fit=crop&auto=format" alt="Tired caregiver | Image credit: butsaya33 – 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 28th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/studies-highlight-heavy-burden-on-caregivers-of-patients-with-dmd">Studies Highlight Heavy Burden on Caregivers of Patients With DMD</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/studies-highlight-heavy-burden-on-caregivers-of-patients-with-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Caregivers of both adults and children with Duchenne muscular dystrophy (DMD) face significant disruptions to their work productivity and personal lives, underscoring the need for better treatments and support systems.</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/zinc-supplements-fall-short-for-boys-with-duchenne-muscular-dystrophy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/ff1a93c1f69b0decd584d3ffa4c1878f8f99c468-7000x4657.jpg?fit=crop&auto=format" alt="Zinc supplement | Image credit: Celt 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 25th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/zinc-supplements-fall-short-for-boys-with-duchenne-muscular-dystrophy">Zinc Supplements Fall Short for Boys With Duchenne Muscular Dystrophy</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/zinc-supplements-fall-short-for-boys-with-duchenne-muscular-dystrophy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Standard zinc supplementation had limited impact on muscle health or zinc levels in boys with Duchenne muscular dystrophy, highlighting the need for more tailored nutritional strategies.</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/genotypes-may-influence-when-patients-with-dmd-have-loss-of-ambulation"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/b2eaf98c1890aeb914d08ef1b39bcf48e888d885-7360x4912.jpg?fit=crop&auto=format" alt="Nurse helping patient use walker | Image credit: LIGHTFIELD STUDIOS – 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/genotypes-may-influence-when-patients-with-dmd-have-loss-of-ambulation">Genotypes May Influence When Patients With DMD Have Loss of Ambulation</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/genotypes-may-influence-when-patients-with-dmd-have-loss-of-ambulation"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A study of 555 patients further solidified genotype-phenotype correlations in Duchenne muscular dystrophy (DMD).</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/wve-n531-shows-promising-efficacy-in-dmd-at-interim-forward-53-analysis"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/6827234f1968e19447a82da682bfa191443aade0-1200x673.jpg?fit=crop&auto=format" alt="Currently, 4 exon-skipping drugs are FDA approved for DMD: eteplirsen (Exondys 51), golodirsen (Vyondys 53), casimersen (Amondys 45), and viltolarsen (Viltepso) | Image credit: CrazyJuke - 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 26th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/wve-n531-shows-promising-efficacy-in-dmd-at-interim-forward-53-analysis">WVE-N531 Shows Promising Efficacy in DMD at Interim FORWARD-53 Analysis</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/wve-n531-shows-promising-efficacy-in-dmd-at-interim-forward-53-analysis"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">In the phase 2 FORWARD-53 study, the exon-skipping oligonucleotide WVE-N531 showed promising safety and efficacy in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.</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/atrial-arrhythmias-common-in-dmd-but-more-data-on-treatment-are-needed"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/716fb1567c89fd0bd7f947c2d2ce88f33b0a3ca0-1200x748.jpg?fit=crop&auto=format" alt="The authors of the case series noted that prevalent comorbidities among patients with DMD increase the risk of arrhythmias even further. | Image credit: Elena - 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 18th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/atrial-arrhythmias-common-in-dmd-but-more-data-on-treatment-are-needed">Atrial Arrhythmias Common in DMD, but More Data on Treatment Are 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/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/atrial-arrhythmias-common-in-dmd-but-more-data-on-treatment-are-needed"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Cases of atrial arrhythmia in patients with Duchenne muscular dystrophy (DMD) are common but lack a standard solution, and more long-term data on the management of arrhythmias in DMD are needed.</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-imaging-algorithm-shows-promise-in-dmd-evaluation"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/256eb619de1c0a8ceade136b979b183b7e6d80d2-1000x667.jpg?fit=crop&auto=format" alt="When there are changes in muscle microstructures, acoustic attenuation occurs, which may hold potential as an imaging biomarker for DMD. | Image credit: hafakot - 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 6th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/novel-imaging-algorithm-shows-promise-in-dmd-evaluation">Novel Imaging Algorithm Shows Promise in DMD Evaluation</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/novel-imaging-algorithm-shows-promise-in-dmd-evaluation"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Measurements on a standard phantom and a clinical data set of patients with Duchenne muscular dystrophy (DMD) were used to validate a novel robust reference frequency method approach, which outperformed typical imaging strategies. </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/payer-education-is-key-to-early-dmd-therapy-access"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/f0799e277f6c57c7cb668ee75d32b229cabf6c5f-1200x640.png?fit=crop&auto=format" alt="Aravindhan Veerapandiyan, MD, a pediatric neuromuscular specialist at Arkansas 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">August 28th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/payer-education-is-key-to-early-dmd-therapy-access">Payer Education Is Key to Early DMD Therapy Access</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/payer-education-is-key-to-early-dmd-therapy-access"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The disease-modifying treatment landscape has expanded in recent years, but access to the latest approved Duchenne muscular dystrophy (DMD) treatments can be a challenge for patients and providers, Aravindhan Veerapandiyan, MD, a pediatric neuromuscular specialist at Arkansas Children’s Hospital, said. </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/cycling-training-shows-protective-effects-on-gait-balance-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg?fit=crop&auto=format" alt="Balance and gait are both important factors in continued mobility among patients with DMD. | Image credit: OlegKachura - 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/cycling-training-shows-protective-effects-on-gait-balance-in-dmd">Cycling Training Shows Protective Effects on Gait, Balance in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/cycling-training-shows-protective-effects-on-gait-balance-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Both cycling and home-based exercise training helped maintain gait and balance parameters in children with Duchenne muscular dystrophy (DMD), with cycling training also improving antero-posterior balance.</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/access-to-novel-dmd-therapies-remains-challenging-despite-regulatory-approvals"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/f0799e277f6c57c7cb668ee75d32b229cabf6c5f-1200x640.png?fit=crop&auto=format" alt="Aravindhan Veerapandiyan, MD, of the Division of Pediatric Neurology at the University of Arkansas for Medical Sciences" 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/access-to-novel-dmd-therapies-remains-challenging-despite-regulatory-approvals">Access to Novel DMD Therapies Remains Challenging Despite Regulatory Approvals</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/access-to-novel-dmd-therapies-remains-challenging-despite-regulatory-approvals"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Aravindhan Veerapandiyan, MD, of the Division of Pediatric Neurology at the University of Arkansas for Medical Sciences, discussed the Duchenne muscular dystrophy (DMD) therapy landscape and barriers to treatment access.</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/significant-pulmonary-upper-limb-function-declines-in-ambulatory-patients-with-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/674b4a7f2c7cd38a9d9029d5d6430eaccac96d0d-5150x3648.jpg?fit=crop&auto=format" alt="The study suggests that ambulation status may not necessarily predict early lung or upper limb function declines in DMD, with some ambulatory patients having early declines in these measures. | Image credti: Nana_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">July 27th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/significant-pulmonary-upper-limb-function-declines-in-ambulatory-patients-with-dmd">Significant Pulmonary, Upper Limb Function Declines in Ambulatory Patients With DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/significant-pulmonary-upper-limb-function-declines-in-ambulatory-patients-with-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Trials of Duchenne muscular dystrophy (DMD) treatments targeting declining lung or upper limb function, which are typically limited to nonambulatory patients, could also include ambulatory patients, according to the study's findings</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/survey-shows-importance-of-discussing-treating-endocrine-effects-of-steroids-for-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/027213521428f14d3c6a035e667845daa4b5c843-1280x904.jpg?fit=crop&auto=format" alt="Duchenne Muscular Dystrophy | 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">July 12th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/survey-shows-importance-of-discussing-treating-endocrine-effects-of-steroids-for-dmd">Survey Shows Importance of Discussing, Treating Endocrine Effects of Steroids for DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/survey-shows-importance-of-discussing-treating-endocrine-effects-of-steroids-for-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Results from a national survey of patients with Duchenne muscular dystrophy (DMD) and their families found that patients and families want to be informed early about endocrine complications associated with glucocorticoid 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 class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/quantifying-the-insurance-value-for-rare-diseases-duchenne-muscular-dystrophy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/5421358c8f117ee86126333e135f08cf1e61ded6-778x382.png?fit=crop&auto=format" alt="AJMC" 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 10th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/quantifying-the-insurance-value-for-rare-diseases-duchenne-muscular-dystrophy">Quantifying the Insurance Value for Rare Diseases: Duchenne Muscular Dystrophy</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jason-shafrin,-phd">Jason Shafrin, PhD</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/suhail-thahir-ms">Suhail Thahir, MS</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/alexa-c-klimchak-ma">Alexa C. Klimchak, MA</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ivana-audhya-msc">Ivana Audhya, MSc</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/lauren-e-sedita-ms">Lauren E. Sedita, MS</a><span class="jsx-ad50481d5ee26850 mr-1 ml-[1px]"> </span><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/john-a-romley-phd">John A. Romley, PhD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/quantifying-the-insurance-value-for-rare-diseases-duchenne-muscular-dystrophy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The degree to which novel value elements such as insurance value impact estimated treatment value for rare, severe genetic diseases such as Duchenne muscular dystrophy is unclear.</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/appendicular-lean-mass-index-fat-mass-index-associated-with-motor-function-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/15941a7b6f8c58f3ee691ab988a2c817fad899b3-7952x5304.jpg?fit=crop&auto=format" alt="DMD, an inherited disorder characterized by progressive muscle weakness, typically manifests in childhood and currently has no cure. | image credit: OlegKachura - 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 5th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/appendicular-lean-mass-index-fat-mass-index-associated-with-motor-function-in-dmd">Appendicular Lean Mass Index, Fat Mass Index Associated With Motor Function in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/rose-mcnulty">Rose McNulty</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/appendicular-lean-mass-index-fat-mass-index-associated-with-motor-function-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Both appendicular lean mass and fat mass index accounted for unique variance in motor function after controlling for age in patients with Duchenne muscular dystrophy (DMD).</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/stem-cell-based-gene-editing-strategy-shows-promise-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/df17be9ab7803f252c60bc1d6eea8586c27dcc08-3898x2598.jpg?fit=crop&auto=format" alt="Gene editing shows promise in Duchenne muscular dystrophy. | Image credit: Degimages - 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">June 29th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/stem-cell-based-gene-editing-strategy-shows-promise-in-dmd">Stem Cell–Based Gene Editing Strategy Shows Promise in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/stem-cell-based-gene-editing-strategy-shows-promise-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein. </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/dmd?page=1">1</a><a class="px-4 py-2 border-y border-r " href="/compendium/dmd?page=2">2</a><a class="px-4 py-2 border-y border-r " href="/compendium/dmd?page=3">3</a><a class="px-4 py-2 border-y border-r" href="/compendium/dmd?page=2">></a></div></div></div><div class="flex-none w-[300px] z-[9999] relative hidden md:block"><div style="top:5rem" class="sticky custom-spacing"><div class="collapse-container " style="overflow:hidden;max-height:900px;transition:max-height .4s ease-in-out"></div></div></div></div><div id="div-gpt-ad-pixel" style="width:1px;height:1px" class=""></div><noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-NK5KQXS" height="0" width="0" 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","published":"2025-02-14T19:58:31.989Z","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"},"authors":[{"displayName":"Rose McNulty","url":"rose-mcnulty"}],"url":"adults-with-dmd-more-than-twice-as-likely-to-experience-fractures","updatedOn":null,"factCheckAuthors":null},{"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"},"authors":[{"displayName":"Rose McNulty","url":"rose-mcnulty"}],"factCheckAuthors":null,"thumbnail":{"_type":"mainImage","alt":"Ifetroban may improve LVEF in patients with DMD. - REDPIXEL - stock.adobe.com","caption":"Ifetroban may improve LVEF in patients with DMD. - REDPIXEL - stock.adobe.com","asset":{"_ref":"image-00882496c7fe122f1e99a913247b7f18619fde44-1200x800-jpg","_type":"reference"}},"summary":"There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results suggest ifetroban may improve left ventricular ejection fraction in patients with DMD. ","updatedOn":null,"_id":"f59d50dc-a45c-430e-ada9-5ec2875d8272","title":"Ifetroban Improves Heart Function in DMD","url":"ifetroban-improves-heart-function-in-dmd","published":"2025-02-07T18:36:00.000Z"},{"_id":"8a635442-33c0-4f89-92dd-449d103bb871","summary":"The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD). ","published":"2025-01-28T14:27:13.510Z","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"},"title":"EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD","url":"embark-2-delandistrogene-moxeparvovec-shows-sustained-benefits-in-dmd","thumbnail":{"_type":"mainImage","alt":"Duchenne muscular dystrophy | Image credit: hafakot - stock.adobe.com","asset":{"_type":"reference","_ref":"image-256eb619de1c0a8ceade136b979b183b7e6d80d2-1000x667-jpg"}},"authors":[{"displayName":"Rose McNulty","url":"rose-mcnulty"}],"factCheckAuthors":null},{"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"},"_id":"5bc95792-f1ef-4e89-80a0-4604bc0996e9","title":"Prophylactic Cardiac Medication May Prolong Survival in DMD","url":"prophylactic-cardiac-medication-may-prolong-survival-in-dmd","summary":"Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals received such treatment, a recent study found. ","published":"2025-01-24T20:00:00.000Z","authors":[{"displayName":"Rose McNulty","url":"rose-mcnulty"}],"factCheckAuthors":null,"thumbnail":{"_type":"mainImage","alt":"Heart and stethoscope | Image credit: inthasone - stock.adobe.com","asset":{"_ref":"image-37d115516d72b70fdccd564ea127cdbc3a31869e-1200x673-jpg","_type":"reference"}},"updatedOn":null},{"authors":[{"displayName":"Jason Shafrin, PhD","url":"jason-shafrin,-phd"},{"displayName":"Suhail Thahir, MS","url":"suhail-thahir-ms"},{"displayName":"Alexa C. Klimchak, MA","url":"alexa-c-klimchak-ma"},{"displayName":"Ivana Audhya, MSc","url":"ivana-audhya-msc"},{"displayName":"Lauren E. Sedita, MS","url":"lauren-e-sedita-ms"},{"displayName":"John A. Romley, PhD","url":"john-a-romley-phd"}],"_id":"3c306f61-7382-428d-af5d-79ff7de945aa","url":"quantifying-the-altruism-value-for-a-rare-pediatric-disease-duchenne-muscular-dystrophy","thumbnail":{"_type":"mainImage","alt":"AJMC","asset":{"_ref":"image-5421358c8f117ee86126333e135f08cf1e61ded6-778x382-png","_type":"reference"}},"factCheckAuthors":null,"title":"Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy","summary":"Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 per year.","published":"2025-01-14T14:00:00.000Z","updatedOn":null,"contentCategory":{"_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh"}},{"_id":"ea3be2e0-e4ed-4f9b-827c-a2b67938d039","thumbnail":{"_type":"mainImage","alt":"Past studies have explored the association between motor and cardiac function in younger patients with DMD, but there is a lack of research on this relationship as patients with DMD age. | Image credit: Nana_studio - stock.adobe.com","caption":"Past studies have explored the association between motor and cardiac function in younger patients with DMD, but there is a lack of research on this relationship as patients with DMD age. | Image credit: Nana_studio - stock.adobe.com","asset":{"_type":"reference","_ref":"image-674b4a7f2c7cd38a9d9029d5d6430eaccac96d0d-5150x3648-jpg"}},"summary":"The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with Duchenne muscular dystrophy (DMD).","published":"2025-01-13T13:05:00.000Z","contentCategory":{"_type":"contentCategory","name":"Articles","_id":"3f4b3ced-7c9d-4fc4-967f-fe993087cce2","_updatedAt":"2023-09-29T14:32:27Z","_createdAt":"2020-04-03T20:03:53Z","_rev":"Q2ZL7ihdIB33NiMMcGccmh"},"title":"Later Loss of Ambulation Linked With Better Heart Health in DMD","url":"later-loss-of-ambulation-linked-with-better-heart-health-in-dmd","updatedOn":null,"authors":[{"displayName":"Rose McNulty","url":"rose-mcnulty"}],"factCheckAuthors":null},{"factCheckAuthors":null,"url":"study-highlights-trends-emerging-innovations-in-dmd-research","published":"2024-12-22T18:23:00.000Z","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"},"_id":"b8f411fd-a4a0-4be6-b7fc-5cf582aa0b30","title":"Study Highlights Trends, Emerging Innovations in DMD Research","thumbnail":{"_type":"mainImage","alt":"lab test tubes | Image credit: kwanchaift – stock.adobe.com","asset":{"_ref":"image-860ecc80e940703fd332d679608fdc354c4efb2b-6016x4016-jpg","_type":"reference"}},"summary":"An analysis revealed global trends and emerging focus areas in Duchenne muscular dystrophy (DMD) research, emphasizing the growing impact of precision medicine and gene therapies.","updatedOn":null,"authors":[{"displayName":"Hayden E. Klein","url":"hayden-e-klein"}]},{"_id":"94f6ad71-750f-4802-b4f9-2332a10edf94","title":"MRI Biomarker May Signal Early Heart Damage in DMD","summary":"Septal circumferential strain (Ecc) was significantly decreased in LGE-negative boys with Duchenne muscular dystrophy (DMD) and correlated to ventricle changes.","published":"2024-12-20T15:35:00.000Z","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,"url":"mri-biomarker-may-signal-early-heart-damage-in-dmd","thumbnail":{"asset":{"_ref":"image-0701c16da7f575b3d0809c6fa104ce4fc03578d6-6000x4000-jpg","_type":"reference"},"_type":"mainImage","alt":"Heart anatomy on paper | Image credit: larisikstefania – stock.adobe.com"},"authors":[{"displayName":"Hayden E. Klein","url":"hayden-e-klein"}]},{"factCheckAuthors":null,"url":"fda-pauses-pepgen-s-dmd-drug-trial-in-the-us","summary":"The CONNECT2-EDO51 phase 2 trial looks at PGN-EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.","published":"2024-12-19T15:51:01.758Z","_id":"69f639fd-1dd5-4138-936b-288a24ddf732","title":"FDA Pauses PepGen’s DMD Drug Trial in the US","thumbnail":{"alt":"Gene therapy | Image credit: Treecha – stock.adobe.com","asset":{"_ref":"image-e9f3330b21f13fe51a104793dc091dacc5848457-5979x3923-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":"Hayden E. Klein","url":"hayden-e-klein"}]},{"_id":"acdd03d4-0f69-41d6-9b2a-ad8525f268fe","url":"top-5-duchenne-muscular-dystrophy-content-of-2024","thumbnail":{"_type":"mainImage","alt":"The top 5 DMD content of 2024","asset":{"_ref":"image-e67fe3af32cb5a8ada28f3f9964afe9005c1ed87-1020x520-png","_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"},"authors":[{"displayName":"Hayden E. Klein","url":"hayden-e-klein"}],"title":"Top 5 Duchenne Muscular Dystrophy Content of 2024","summary":"From imaging innovations to new treatment approvals, 2024 brought advancements in Duchenne muscular dystrophy care and research.","published":"2024-12-19T12:36:00.000Z","factCheckAuthors":null},{"updatedOn":null,"_id":"ee73b7bd-b489-4b0b-895b-1cf321d48b3a","title":"Real-World Challenges in DMD PMO 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":"Hayden E. Klein","url":"hayden-e-klein"}],"factCheckAuthors":null,"url":"real-world-challenges-in-dmd-pmo-treatment","thumbnail":{"_type":"mainImage","alt":"Gene therapy graphic with DNA strands | Image credit: natali_mis – stock.adobe.com","asset":{"_ref":"image-d2c61cb6916c5df55b1355585d6bf4a6bf23fbce-6720x4480-jpg","_type":"reference"}},"summary":"A claims-based study revealed high adherence to phosphorodiamidate morpholino oligomer (PMO) treatments for Duchenne muscular dystrophy (DMD), but highlights limitations in interpreting real-world effectiveness from claims data.","published":"2024-11-25T17:30:08.312Z"},{"thumbnail":{"alt":"Boy with DMD using walker | Image credit: Svitlana – stock.adobe.com","asset":{"_ref":"image-7e20ff65b7abea493e84dace957fb80991ce7bb1-6048x4024-jpg","_type":"reference"},"_type":"mainImage"},"published":"2024-11-23T18:00:00.000Z","factCheckAuthors":null,"summary":"Further research is needed to determine how once-weekly viltolarsen can slow disease progression and preserve motor function in boys and young men with Duchenne muscular dystrophy (DMD).","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"},"authors":[{"displayName":"Hayden E. Klein","url":"hayden-e-klein"}],"_id":"86d3362c-b948-469e-80be-ca1af7693cbe","title":"Viltolarsen Slows DMD Progression in Small Study","url":"viltolarsen-slows-dmd-progression-in-small-study"},{"factCheckAuthors":null,"_id":"fdd70e9f-b0c6-4be2-91ec-f6b65108cec6","title":"FDA Accepts Resubmitted NDA for Ataluren in Nonsense Duchenne Muscular Dystrophy","thumbnail":{"_type":"mainImage","alt":"FDA website | Image Credit: © Postmodern Studio - stock.adobe.com","asset":{"_ref":"image-ebfb40d061db8cb94776018fe5f4c50f7871539b-5293x3529-jpg","_type":"reference"}},"summary":"The new drug application (NDA) includes data from a global placebo-controlled, 72-week study as well as findings from the STRIDE registry, an ongoing, observational, real-world study of ataluren in routine care.","updatedOn":null,"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"},"url":"fda-accepts-resubmitted-nda-for-ataluren-in-nonsense-duchenne-muscular-dystrophy","published":"2024-11-22T21:25:00.000Z","authors":[{"displayName":"Marco Meglio","url":"marco-meglio"}]},{"_id":"11347787-4ff4-428c-863c-9ea85d172939","url":"sarepta-halts-development-testing-of-dmd-therapy-srp-5051","thumbnail":{"_type":"mainImage","alt":"Researcher working with microplate in the laboratory | Image credit: angellodeco – stock.adobe.com","asset":{"_ref":"image-a7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648-jpg","_type":"reference"}},"summary":"Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with the developer citing safety concerns, FDA feedback, and an evolving therapeutic landscape.","published":"2024-11-14T16:35:33.459Z","updatedOn":null,"authors":[{"displayName":"Hayden E. Klein","url":"hayden-e-klein"}],"title":"Sarepta Halts Development, Testing of DMD Therapy SRP-5051","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":"f8d65f6f-fd6c-4dc7-a3bb-8816b9faeafd","url":"a-year-of-dmd-gene-therapy-trial-failures","thumbnail":{"asset":{"_ref":"image-f17e24452d6c5da3f5c3add1df744b5a59621ffe-6000x4000-jpg","_type":"reference"},"_type":"mainImage","alt":"Gene therapy research | Image credit: RFBSIP – stock.adobe.com"},"published":"2024-10-29T18:27:00.000Z","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":"Hayden E. Klein","url":"hayden-e-klein"}],"title":"A Year of DMD Gene Therapy Trial Failures","summary":"The outcomes of the EMBARK and CIFFREO trials in boys aged 4 to 7 have raised concerns in the Duchenne muscular dystrophy (DMD) community and prompted layoffs at Pfizer.","updatedOn":"2024-10-30T14:52:39.327Z","factCheckAuthors":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"},"factCheckAuthors":null,"title":"Studies Highlight Heavy Burden on Caregivers of Patients With DMD","summary":"Caregivers of both adults and children with Duchenne muscular dystrophy (DMD) face significant disruptions to their work productivity and personal lives, underscoring the need for better treatments and support systems.","authors":[{"displayName":"Hayden E. Klein","url":"hayden-e-klein"}],"_id":"c45ce419-bc4d-450c-8c10-2c3039ac6bc0","url":"studies-highlight-heavy-burden-on-caregivers-of-patients-with-dmd","thumbnail":{"alt":"Tired caregiver | Image credit: butsaya33 – stock.adobe.com","asset":{"_ref":"image-7b01dbf92510da0d1d91407c2370108cc29fa2ac-5472x3648-jpg","_type":"reference"},"_type":"mainImage"},"published":"2024-10-28T14:57:13.055Z","updatedOn":null},{"authors":[{"displayName":"Hayden E. 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And oftentimes, [we're] trying to find a way of replacing the full length of the dystrophin gene, as opposed to micro-dystrophin or mini-dystrophin—so, finding alternative methods of having perhaps viral vectors that could replace parts of it, and then they'll assemble back together intracellularly again, or finding other ways of having gene therapy delivered without viral vectors that don't activate the immune system.","_key":"910e88bf611f"}]},{"_key":"09a23ae955e1","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"be13c8b5b82e"}],"_type":"block","style":"normal"},{"_type":"block","style":"normal","_key":"8910913a8ef0","markDefs":[],"children":[{"_key":"e5a69d733055","_type":"span","marks":[],"text":"I think those will be areas for future research that are really, really promising, as well as other combinations of therapies—not just the steroids, but nonsteroidal things at the cellular level, working on regenerating muscle for the patients that have already had some of that muscle [loss] before they receive their gene therapy. 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We can't say it's a cure, because you're not replacing the full length of the dystrophin protein, but hopefully, it will be enough to halt the disease progress and it can be sustained over the years. 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