News — The Stop ALD Foundation

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But it is urgent that we invest in research that will advance new therapies, as well as new tools for early diagnosis and educational campaigns that will raise awareness so that children with ALD can be identified as early as possible, while there is the best hope of  a beneficial treatment.</p><p>Advancing such research is the goal of the Stop ALD Foundation. Like a venture capital firm, we identify gaps in the development process, seek out creative solutions to fill those gaps, and deliver the resources that can develop these great ideas into concrete projects, and projects into therapies that can transform the lives of children with ALD and their families.</p>","image":"//images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1395608857481-B450M7Q6MBY0IP5B98EW/TheSTOP++ALD+Foundation+Logo.jpg","@context":"http://schema.org","@type":"WebSite"}</script><script type="application/ld+json">{"legalName":"The Stop ALD Foundation","address":" Suite 2000\nHouston, TX 77002\nUSA","email":"info@stopald.org","telephone":"+1.713.756.3232","sameAs":["http://www.facebook.com/stopald","https://twitter.com/StopALD"],"@context":"http://schema.org","@type":"Organization"}</script><script type="application/ld+json">{"address":" Suite 2000\nHouston, TX 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hfeed"> <article class="hentry author-steve-barsh post-type-text article-index-1" id="article-6671073024a0c62bfdf9672d" data-item-id="6671073024a0c62bfdf9672d">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2024/6/18/leriglitazone-halts-disease-progression-in-adult-patients-with-early-cerebral-adrenoleukodystrophy-ald-in-compassionate-use-study-published-in-brain">Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy (ALD) in compassionate use study published in Brain</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2024-06-18" itemprop="datePublished" pubdate><a href="/news-1/2024/6/18/leriglitazone-halts-disease-progression-in-adult-patients-with-early-cerebral-adrenoleukodystrophy-ald-in-compassionate-use-study-published-in-brain">June 18, 2024</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1718683576360" id="item-6671073024a0c62bfdf9672d"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-c09e468088322f20b85a"><div class="sqs-block-content"> <div class="sqs-html-content"> <p class="" style="white-space:pre-wrap;"><strong>In a French early-access program study, 10 of 13 patients were clinically and radiologically stable</strong></p><p class="" style="white-space:pre-wrap;"><strong>Mataró, Barcelona, Spain, 11 June, 2024</strong> - <a href="https://www.minoryx.com/">Minoryx Therapeutics</a> (“Minoryx”), a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announce that results from a compassionate use study of <a href="https://www.minoryx.com/leriglitazone/">leriglitazone</a> for treatment of progressive cerebral adrenoleukodystrophy (cALD) in adult male patients, <a href="https://academic.oup.com/brain/advance-article-abstract/doi/10.1093/brain/awae169/7687562?redirectedFrom=fulltext">published</a>1 in the peer-reviewed journal <em>Brain</em>.</p><p class="" style="white-space:pre-wrap;">The compassionate-use study was led by Fanny Mochel, MD, Ph.D., professor at the Hôspital Universitaire La Pitié-Salpêtrière (Paris, France), as part of an early-access program through the French national drug agency (ANSM) that allows for the use of innovative treatments with favorable safety profiles under a Compassionate Access Authorization protocol (ATU)2. <a href="https://www.pharmiweb.com/press-release/2024-06-14/leriglitazone-halts-disease-progression-in-adult-patients-with-early-cerebral-adrenoleukodystrophy-in-compassionate-use-study-published-in-brain" target="_blank">More…</a></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2024/6/18/leriglitazone-halts-disease-progression-in-adult-patients-with-early-cerebral-adrenoleukodystrophy-ald-in-compassionate-use-study-published-in-brain#comments-6671073024a0c62bfdf9672d">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/6671073024a0c62bfdf9672d/1718683576573/" data-record-type="1" data-full-url="/news-1/2024/6/18/leriglitazone-halts-disease-progression-in-adult-patients-with-early-cerebral-adrenoleukodystrophy-ald-in-compassionate-use-study-published-in-brain" data-title="Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy (ALD) in compassionate use study published in Brain"></span> <span class="sqs-simple-like" data-item-id="6671073024a0c62bfdf9672d" data-like-count="1"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-2" id="article-64776c9eedf3993ace9e5399" data-item-id="64776c9eedf3993ace9e5399">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2023/5/31/minoryx-gains-fda-approval-to-initiate-a-phase-3-clinical-trial-in-patients-with-cerebral-adrenoleukodystrophy">Minoryx gains FDA approval to initiate a Phase 3 clinical trial in patients with cerebral Adrenoleukodystrophy</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2023-05-31" itemprop="datePublished" pubdate><a href="/news-1/2023/5/31/minoryx-gains-fda-approval-to-initiate-a-phase-3-clinical-trial-in-patients-with-cerebral-adrenoleukodystrophy">May 31, 2023</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1685548339322" id="item-64776c9eedf3993ace9e5399"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" data-border-radii="{"topLeft":{"unit":"px","value":0.0},"topRight":{"unit":"px","value":0.0},"bottomLeft":{"unit":"px","value":0.0},"bottomRight":{"unit":"px","value":0.0}}" id="block-981c9f35e0b6f239d2f9"><div class="sqs-block-content"> <div class="sqs-html-content"> <p class="" style="white-space:pre-wrap;"><strong>US trial with leriglitazone to commence in mid-2023 providing an FDA-approved route to US market whilst EMA evaluates European Marketing Authorization Application (MAA)</strong></p><p class="" style="white-space:pre-wrap;">Mataró, Barcelona, Spain, May 31, 2023 - <a href="https://www.minoryx.com" target="_blank">Minoryx Therapeutics</a>, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the US Food and Drug Administration (FDA) approval of its Phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male <a href="https://www.minoryx.com/adrenoleukodystrophy-orphan-disease/" target="_blank">X-linked Adrenoleukodystrophy (X-ALD)</a> patients with cerebral Adrenoleukodystrophy (cALD). <a href="https://www.minoryx.com/media/minoryx-gains-fda-approval-to-initiate-a-phase-3-clinical-trial-in-patients-with-cerebral-adrenoleukodystrophy/" target="_blank">More…</a></p><p class="" data-rte-preserve-empty="true" style="white-space:pre-wrap;"></p><p class="" data-rte-preserve-empty="true" style="white-space:pre-wrap;"></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2023/5/31/minoryx-gains-fda-approval-to-initiate-a-phase-3-clinical-trial-in-patients-with-cerebral-adrenoleukodystrophy#comments-64776c9eedf3993ace9e5399">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/64776c9eedf3993ace9e5399/1685548339459/" data-record-type="1" data-full-url="/news-1/2023/5/31/minoryx-gains-fda-approval-to-initiate-a-phase-3-clinical-trial-in-patients-with-cerebral-adrenoleukodystrophy" data-title="Minoryx gains FDA approval to initiate a Phase 3 clinical trial in patients with cerebral Adrenoleukodystrophy"></span> <span class="sqs-simple-like" data-item-id="64776c9eedf3993ace9e5399" data-like-count="1"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-3" id="article-63fc1d578dfb7d202afd4892" data-item-id="63fc1d578dfb7d202afd4892">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2023/2/26/minoryx-therapeutics-announces-x-linked-adrenoleukodystrophy-data">Minoryx Therapeutics announces X-linked adrenoleukodystrophy data</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2023-02-26" itemprop="datePublished" pubdate><a href="/news-1/2023/2/26/minoryx-therapeutics-announces-x-linked-adrenoleukodystrophy-data">February 26, 2023</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1677467321879" id="item-63fc1d578dfb7d202afd4892"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" data-border-radii="{"topLeft":{"unit":"px","value":0.0},"topRight":{"unit":"px","value":0.0},"bottomLeft":{"unit":"px","value":0.0},"bottomRight":{"unit":"px","value":0.0}}" id="block-7ec5abc4dd3faab15402"><div class="sqs-block-content"> <div class="sqs-html-content"> <p class="" style="white-space:pre-wrap;"><strong>Minoryx Therapeutics has announced that data from its phase 2/3 ADVANCE clinical trial of lead candidate, leriglitazone, has been </strong><a href="https://www.pharmiweb.com/press-release/2023-01-20/the-lancet-neurology-publishes-results-from-minoryx-therapeutics-phase-23-advance-clinical-trial-of-leriglitazone-in-x-linked-adrenoleukodystrophy" target="_blank"><strong>published in The Lancet Neurology</strong></a><strong>.</strong></p><p class="" style="white-space:pre-wrap;">The trial, assessing the efficacy and safety of leriglitazone in male patients with adrenomyeloneuropathy, is the first and largest international study to enroll adult male X-linked adrenoleukodystrophy patients. </p><p class="" style="white-space:pre-wrap;"><a href="https://www.labiotech.eu/trends-news/minoryx-therapeutics-x-linked-adrenoleukodystrophy-data/" target="_blank">More…</a></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2023/2/26/minoryx-therapeutics-announces-x-linked-adrenoleukodystrophy-data#comments-63fc1d578dfb7d202afd4892">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/63fc1d578dfb7d202afd4892/1677467790786/" data-record-type="1" data-full-url="/news-1/2023/2/26/minoryx-therapeutics-announces-x-linked-adrenoleukodystrophy-data" data-title="Minoryx Therapeutics announces X-linked adrenoleukodystrophy data"></span> <span class="sqs-simple-like" data-item-id="63fc1d578dfb7d202afd4892" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-4" id="article-63fc202f4f1b05297e358b5e" data-item-id="63fc202f4f1b05297e358b5e">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2023/2/26/stanford-research-study-on-vitamin-d-needs-input-from-parents-or-guardians-of-boys-with-ald">Stanford Research Study on Vitamin D Needs Input from Parents or Guardians of Boys with ALD</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2023-02-26" itemprop="datePublished" pubdate><a href="/news-1/2023/2/26/stanford-research-study-on-vitamin-d-needs-input-from-parents-or-guardians-of-boys-with-ald">February 26, 2023</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1677467695701" id="item-63fc202f4f1b05297e358b5e"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" data-border-radii="{"topLeft":{"unit":"px","value":0.0},"topRight":{"unit":"px","value":0.0},"bottomLeft":{"unit":"px","value":0.0},"bottomRight":{"unit":"px","value":0.0}}" id="block-b5ec4c81e72bc6c9280b"><div class="sqs-block-content"> <div class="sqs-html-content"> <p class="" style="white-space:pre-wrap;">Attention ALD Families: A Stanford Research Study on Vitamin D needs the input of parents or guardians of boys with ALD. This survey is essential to design a vitamin D study that is in line with the values and interests of ALD families. </p><p class="" style="white-space:pre-wrap;">You can complete the survey <a href="https://l.facebook.com/l.php?__tn__=%2CmH-R&c%5B0%5D=AT1tSJoffzEFwg6SyAn8yypE63rs4yFjblvq1JLjZHZKQNJs3iJjpa3ujIJOIvukiFwRIqUY5UsgQlt3LU0689R4ZJTKdLIIDAI9ojrbFSj6WxiOZxjVaHkS9EZQL8pxfpOyD9t4JfoE1i-duCqTxUG6QHjw4hpLKRjt4AMYWhbgYCgRDDyXBNkzVoCUDPOzV95uILoOpUf-&h=AT0aug9CrBPCL0TPzBQ7zLXdpUY_nRiQVe0lT8uc95aUALmg3TBDGd7vkJuDJMwNwFjlimJSYaBzrUoatHozl90z1Pi1hM6VAqY4uwXc1tXfrZ_nJeUmzXxLFjNwfjDvKdpJr4g&u=http%3A%2F%2Ff1ahhllmfjr.typeform.com%2Fto%2FClnDtZXR%3Ffbclid%3DIwAR24U20uHqSVzJyM6M0YiOh0uOPoA74YHUHIThKsBpJ2o3W1mzZzrIa6ixs" target="_blank">here</a>.</p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2023/2/26/stanford-research-study-on-vitamin-d-needs-input-from-parents-or-guardians-of-boys-with-ald#comments-63fc202f4f1b05297e358b5e">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/63fc202f4f1b05297e358b5e/1677467695907/" data-record-type="1" data-full-url="/news-1/2023/2/26/stanford-research-study-on-vitamin-d-needs-input-from-parents-or-guardians-of-boys-with-ald" data-title="Stanford Research Study on Vitamin D Needs Input from Parents or Guardians of Boys with ALD"></span> <span class="sqs-simple-like" data-item-id="63fc202f4f1b05297e358b5e" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-5" id="article-601773eec8d9b527a250667a" data-item-id="601773eec8d9b527a250667a">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2021/1/31/minoryx-presents-topline-results-from-phase-23-study-demonstrating-significant-clinical-benefit-for-adrenomyeloneuropathy-amn">Minoryx presents topline results from Phase 2/3 study demonstrating significant clinical benefit for adrenomyeloneuropathy (AMN)</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2021-01-31" itemprop="datePublished" pubdate><a href="/news-1/2021/1/31/minoryx-presents-topline-results-from-phase-23-study-demonstrating-significant-clinical-benefit-for-adrenomyeloneuropathy-amn">January 31, 2021</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1612149952695" id="item-601773eec8d9b527a250667a"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-84960dc94cf22fd3a817"><div class="sqs-block-content"> <div class="sqs-html-content"> <p class="" style="white-space:pre-wrap;"><strong>Largest prospective efficacy study conducted in the orphan disease AMN</strong></p><ul data-rte-list="default"><li><p class="" style="white-space:pre-wrap;"><strong>Leriglitazone significantly reduces progression of cerebral lesions and myelopathy symptoms</strong></p></li><li><p class="" style="white-space:pre-wrap;"><strong>Minoryx preparing for discussions with regulators for approval of leriglitazone for AMN patients</strong></p></li></ul><p class="" style="white-space:pre-wrap;"><strong>Mataró, Barcelona, Spain, January 26, 2021</strong> - Minoryx Therapeutics, a Phase 3 clinical stage biotech company focused on the development of differentiating treatment options in orphan central nervous system (CNS) disorders, today announces topline results from its Phase 2/3 ADVANCE clinical trial. The study evaluated leriglitazone, a novel, selective PPARγ agonist, in male patients with adrenomyeloneuropathy (AMN), a neurodegenerative disease causing progressive spastic paraparesis and autonomic nervous system dysfunction. Additionally, AMN patients are at risk of developing progressive cerebral lesions, a devastating form of the disease leading to rapid severe disability and fatal if left untreated.</p><p class="" style="white-space:pre-wrap;"><a href="https://www.minoryx.com/media/minoryx_presents_topline_results_from_phase_2-3_%E2%80%9Cadvance%E2%80%9D_study_demonstrating_significant_clinical_benefit_of_leriglitazone_in_adrenomyeloneuropathy_(amn)/" target="_blank">More…</a></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2021/1/31/minoryx-presents-topline-results-from-phase-23-study-demonstrating-significant-clinical-benefit-for-adrenomyeloneuropathy-amn#comments-601773eec8d9b527a250667a">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/601773eec8d9b527a250667a/1612149952876/" data-record-type="1" data-full-url="/news-1/2021/1/31/minoryx-presents-topline-results-from-phase-23-study-demonstrating-significant-clinical-benefit-for-adrenomyeloneuropathy-amn" data-title="Minoryx presents topline results from Phase 2/3 study demonstrating significant clinical benefit for adrenomyeloneuropathy (AMN)"></span> <span class="sqs-simple-like" data-item-id="601773eec8d9b527a250667a" data-like-count="2"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry promoted promoted-block-image author-steve-barsh post-type-text article-index-6" id="article-5dfeaf5550c5304730165d18" data-item-id="5dfeaf5550c5304730165d18">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2019/12/21/stanford-medicine-survey-for-x-ald-caregivers-due-by-feb-28-2020">Stanford Medicine Survey for X-ALD Caregivers (due by Feb 28, 2020)</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2019-12-21" itemprop="datePublished" pubdate><a href="/news-1/2019/12/21/stanford-medicine-survey-for-x-ald-caregivers-due-by-feb-28-2020">December 21, 2019</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>  <div class="special-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Promoted Block" data-type="promoted-block" id="promoted-block-5dfeaf5550c5304730165d18"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block image-block sqs-block-image" data-aspect-ratio="25.080906148867317" data-block-type="5" id="block-yui_3_17_2_1_1576972103170_18051"><div class="sqs-block-content"> <div class=" image-block-outer-wrapper layout-caption-hidden design-layout-inline combination-animation-none individual-animation-none individual-text-animation-none " data-test="image-block-inline-outer-wrapper" > <figure class=" sqs-block-image-figure intrinsic " style="max-width:390px;" > <div class="image-block-wrapper" data-animation-role="image" > <div class="sqs-image-shape-container-element has-aspect-ratio " style=" position: relative; padding-bottom:25.08090591430664%; overflow: hidden;-webkit-mask-image: -webkit-radial-gradient(white, black); " > <img data-stretch="false" data-src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png" data-image="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png" data-image-dimensions="390x129" data-image-focal-point="0.5,0.5" alt="download.png" data-load="false" elementtiming="system-image-block" src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png" width="390" height="129" alt="" sizes="(max-width: 640px) 100vw, (max-width: 767px) 100vw, 100vw" style="display:block;object-fit: cover; width: 100%; height: 100%; object-position: 50% 50%" onload="this.classList.add("loaded")" srcset="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png?format=100w 100w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png?format=300w 300w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png?format=500w 500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png?format=750w 750w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png?format=1000w 1000w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png?format=1500w 1500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1576972251602-98Z8H519257TYVPRD74Q/download.png?format=2500w 2500w" loading="lazy" decoding="async" data-loader="sqs"> </div> </div> </figure> </div> </div></div></div></div></div></div>  <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1576972466647" id="item-5dfeaf5550c5304730165d18"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-e81be450ce7b0c9765dd"><div class="sqs-block-content"> <div class="sqs-html-content"> <p class="" style="white-space:pre-wrap;">Do you care for a child who had a positive newborn screen for X-linked adrenoleukodystrophy (X-ALD)?</p><p class="" style="white-space:pre-wrap;">If so, please consider <a href="https://tinyurl.com/y6jx7ht7">participating in a <strong>one-time online survey</strong>.</a> </p><p class="" style="white-space:pre-wrap;">The goal of the study is to help healthcare professionals better understand the quality of life for caregivers of children with a positive X-ALD newborn screen. In the future we hope to use this information to better meet caregivers needs. For additional information and <strong>to </strong><a href="https://tinyurl.com/y6jx7ht7"><strong>access the free online survey</strong></a><strong> , please visit </strong><a href="https://tinyurl.com/y6jx7ht7 "><strong>https://tinyurl.com/y6jx7ht7</strong></a><strong> </strong></p><p class="" style="white-space:pre-wrap;">The online survey will be available <strong>until February 28, 2020</strong>. Questions? Please email the study director, Kayla Muirhead, B.S., at kaylajm@stanford.edu. Participant's rights questions, contact 1-866-680-2906. </p><p class="" style="white-space:pre-wrap;"><strong>X-linked Adrenoleukodystrophy (X-ALD) caregiver study seeking participants! </strong></p><p class="" style="white-space:pre-wrap;">The study consists of a <strong>one- time online survey </strong>for caregivers of children who screen positive for X-ALD on newborn screening. The questions in the survey ask about basic demographic information and caregiver quality of life. It is estimated to take about 10-20 minutes to complete. </p><p class="" style="white-space:pre-wrap;"><strong>What are the goals of the study? </strong></p><ul data-rte-list="default"><li><p class="" style="white-space:pre-wrap;">Measure quality of life for caregivers.</p></li><li><p class="" style="white-space:pre-wrap;">Suggest factors that influence caregiver quality of life.</p></li><li><p class="" style="white-space:pre-wrap;">Use the information from this study to help providers better care for families with X-ALD. </p></li></ul><p class="" style="white-space:pre-wrap;"><strong>What can participants of the X-ALD caregiver study expect? </strong></p><p class="" style="white-space:pre-wrap;">Caregivers will be asked to: </p><ol data-rte-list="default"><li><p class="" style="white-space:pre-wrap;">Provide their child’s newborn screening results to study personnel </p></li><li><p class="" style="white-space:pre-wrap;">Answer a series of questions on the 10-20 minute one-time online survey that measure quality of life and give providers additional background information </p></li></ol><p class="" style="white-space:pre-wrap;"><strong>Why participate in the X-ALD caregiver study? </strong></p><p class="" style="white-space:pre-wrap;">This information will help doctors and healthcare providers care for affected individuals and their families and will be used to inform ways to improve overall quality of life. </p><p class="" style="white-space:pre-wrap;">There will be no cost to families associated with participating in this study. If you provide your contact information, you will be entered into a raffle to be conducted on March 2, 2020 (3 days after the survey is closed) for a $50 Amazon gift card. The drawing will be conducted by Kayla Muirhead in Palo Alto, California. Participation in the study is not required in order to participate in the raffle. You can enter the raffle if you do not start or complete the study task. The chance of winning a prize is approximately 1 in 50. The winner will be notified immediately by email and provided with information on how to receive the prize. </p><p class="" style="white-space:pre-wrap;"><strong>Who can participate in the X-ALD caregiver study? </strong></p><p class="" style="white-space:pre-wrap;">Any caregiver who has a child with a confirmed diagnosis of X-ALD found on newborn screening in the states of California or New York. </p><p class="" style="white-space:pre-wrap;"><strong>Interested in participating in the study? </strong></p><p class="" style="white-space:pre-wrap;">Access the one-time online survey at <strong>https://tinyurl.com/y6jx7ht7 </strong>(survey available until February 28, 2020). </p><p class="" style="white-space:pre-wrap;">The Principal Investigator of the study is Kayla Muirhead, B.S. To learn more about the study or for inquiries, please contact Kayla Muirhead at kaylajm@stanford.edu. Participant's rights questions, contact 1-866-680-2906. </p><p class="" style="white-space:pre-wrap;">*If you provide your contact information, you will be entered into a raffle to be conducted on March 2, 2020 (3 days after the survey is closed) for a $50 Amazon gift card. The drawing will be conducted by Kayla Muirhead in Palo Alto, California. Participation in the study is not required in order to participate in the raffle. You can enter the raffle if you do not start or complete the study task. The chance of winning a prize is approximately 1 in 50. The winner will be notified immediately by email and provided with information on how to receive the prize. </p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2019/12/21/stanford-medicine-survey-for-x-ald-caregivers-due-by-feb-28-2020#comments-5dfeaf5550c5304730165d18">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/5dfeaf5550c5304730165d18/1576972466999/" data-record-type="1" data-full-url="/news-1/2019/12/21/stanford-medicine-survey-for-x-ald-caregivers-due-by-feb-28-2020" data-title="Stanford Medicine Survey for X-ALD Caregivers (due by Feb 28, 2020)"></span> <span class="sqs-simple-like" data-item-id="5dfeaf5550c5304730165d18" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-7" id="article-5d739f82f7fc2410b6a8b3b5" data-item-id="5d739f82f7fc2410b6a8b3b5">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2019/9/7/ald-newborn-screening-now-in-texas">ALD Newborn Screening Now in Texas!</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2019-09-07" itemprop="datePublished" pubdate><a href="/news-1/2019/9/7/ald-newborn-screening-now-in-texas">September 7, 2019</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1567858684173" id="item-5d739f82f7fc2410b6a8b3b5"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-400390e069b2e034a886"><div class="sqs-block-content"> <div class="sqs-html-content"> <h1 style="white-space:pre-wrap;">It took a village to achieve’: Texas’ newborn screening lab now testing for ALD</h1> </div> </div></div><div class="sqs-block image-block sqs-block-image" data-block-type="5" id="block-yui_3_17_2_1_1567858701480_7341"><div class="sqs-block-content"> <div class=" image-block-outer-wrapper layout-caption-below design-layout-inline combination-animation-none individual-animation-none individual-text-animation-none " data-test="image-block-inline-outer-wrapper" > <figure class=" sqs-block-image-figure intrinsic " style="max-width:824px;" > <div class="image-block-wrapper" data-animation-role="image" > <div class="sqs-image-shape-container-element has-aspect-ratio " style=" position: relative; padding-bottom:56.31067657470703%; overflow: hidden;-webkit-mask-image: -webkit-radial-gradient(white, black); " > <img data-stretch="false" data-src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png" data-image="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png" data-image-dimensions="824x464" data-image-focal-point="0.5,0.5" alt="ribbon-cutting.png" data-load="false" elementtiming="system-image-block" src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png" width="824" height="464" alt="" sizes="(max-width: 640px) 100vw, (max-width: 767px) 100vw, 100vw" style="display:block;object-fit: cover; width: 100%; height: 100%; object-position: 50% 50%" onload="this.classList.add("loaded")" srcset="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png?format=100w 100w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png?format=300w 300w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png?format=500w 500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png?format=750w 750w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png?format=1000w 1000w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png?format=1500w 1500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1567858841970-1BWHOTHUC5BQYXYHUV2O/ribbon-cutting.png?format=2500w 2500w" loading="lazy" decoding="async" data-loader="sqs"> </div> </div> </figure> </div> </div></div><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-yui_3_17_2_1_1567858701480_7618"><div class="sqs-block-content"> <div class="sqs-html-content"> <p class="" style="white-space:pre-wrap;">AUSTIN (Nexstar) — Newborn babies in Texas are <a href="https://www.kxan.com/news/simplehealth/newborn-babies-in-texas-will-soon-be-tested-for-ald/">now getting tested for adrenoleukodystrophy, commonly known as ALD.</a> </p><p class="" style="white-space:pre-wrap;">Texas joins 15 other states in testing for this condition in every baby born in the state. </p><p class="" style="white-space:pre-wrap;">The rare genetic disorder occurs primarily in males and affects the nervous system, as well as the adrenal glands. It impacts around one in every 18,000 people. </p><p class="" style="white-space:pre-wrap;">State health officials, legislators and parents toured the Texas Department of State Health Services’ newborn screening laboratory to celebrate the addition Thursday.</p><p class="" style="white-space:pre-wrap;"><a href="https://www.bigcountryhomepage.com/news/it-took-a-village-to-achieve-texas-newborn-screening-lab-now-testing-for-ald/" target="_blank">Read more…</a></p><p class="" style="white-space:pre-wrap;"><br><br><br></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2019/9/7/ald-newborn-screening-now-in-texas#comments-5d739f82f7fc2410b6a8b3b5">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/5d739f82f7fc2410b6a8b3b5/1567858894506/" data-record-type="1" data-full-url="/news-1/2019/9/7/ald-newborn-screening-now-in-texas" data-title="ALD Newborn Screening Now in Texas!"></span> <span class="sqs-simple-like" data-item-id="5d739f82f7fc2410b6a8b3b5" data-like-count="1"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-8" id="article-5d005a93f5411700013c2ead" data-item-id="5d005a93f5411700013c2ead">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2019/6/11/biomarker-identification-safety-and-efficacy-of-high-dose-antioxidants-for-adrenomyeloneuropathy-amn-a-phase-ii-pilot-study">Biomarker Identification, Safety, and Efficacy of High-Dose Antioxidants for Adrenomyeloneuropathy (AMN): a Phase II Pilot Study</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2019-06-11" itemprop="datePublished" pubdate><a href="/news-1/2019/6/11/biomarker-identification-safety-and-efficacy-of-high-dose-antioxidants-for-adrenomyeloneuropathy-amn-a-phase-ii-pilot-study">June 11, 2019</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1560304346178" id="item-5d005a93f5411700013c2ead"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-b5672bff113316f3f847"><div class="sqs-block-content"> <div class="sqs-html-content"> <h2 style="white-space:pre-wrap;">Abstract</h2><p class="" style="white-space:pre-wrap;">X-Adrenoleukodystrophy (X-ALD) and its adult-onset, most prevalent variant adrenomyeloneuropathy (AMN) are caused by mutations in the peroxisomal transporter of the very long-chain fatty acid ABCD1. AMN patients classically present spastic paraparesis that can progress over decades, and a satisfactory treatment is currently lacking. Oxidative stress is an early culprit in X-ALD pathogenesis. A combination of antioxidants halts the clinical progression and axonal damage in a murine model of AMN, providing a strong rationale for clinical translation. In this phase II pilot, open-label study, 13 subjects with AMN were administered a high dose of α-tocopherol, N-acetylcysteine, and α-lipoic acid in combination. The primary outcome was the validation of a set of biomarkers for monitoring the biological effects of this and future treatments. Functional clinical scales, the 6-minute walk test (6MWT), electrophysiological studies, and cerebral MRI served as secondary outcomes. Most biomarkers of oxidative damage and inflammation were normalized upon treatment, indicating an interlinked redox and inflammatory homeostasis. Two of the inflammatory markers, MCP1 and 15-HETE, were predictive of the response to treatment. We also observed a significant decrease in central motor conduction time, together with an improvement or stabilization of the 6MWT in 8/10 subjects. This study provides a series of biomarkers that are useful to monitor redox and pro-inflammatory target engagement in future trials, together with candidate biomarkers that may serve for patient stratification and disease progression, which merit replication in future clinical trials. Moreover, the clinical results suggest a positive signal for extending these studies to phase III randomized, placebo-controlled, longer-term trials with the actual identified dose. <a href="http://clinicaltrials.gov/" target="_blank"><span style="text-decoration:underline">ClinicalTrials.gov</span></a> Identifier: NCT01495260</p><p class="" style="white-space:pre-wrap;"><a href="https://link.springer.com/article/10.1007%2Fs13311-019-00735-2" target="_blank">Read more…</a></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2019/6/11/biomarker-identification-safety-and-efficacy-of-high-dose-antioxidants-for-adrenomyeloneuropathy-amn-a-phase-ii-pilot-study#comments-5d005a93f5411700013c2ead">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/5d005a93f5411700013c2ead/1560304346306/" data-record-type="1" data-full-url="/news-1/2019/6/11/biomarker-identification-safety-and-efficacy-of-high-dose-antioxidants-for-adrenomyeloneuropathy-amn-a-phase-ii-pilot-study" data-title="Biomarker Identification, Safety, and Efficacy of High-Dose Antioxidants for Adrenomyeloneuropathy (AMN): a Phase II Pilot Study"></span> <span class="sqs-simple-like" data-item-id="5d005a93f5411700013c2ead" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry promoted promoted-block-image author-steve-barsh post-type-text article-index-9" id="article-5bdad323b8a0458599c6aedb" data-item-id="5bdad323b8a0458599c6aedb">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2018/11/1/families-in-california-who-received-a-positive-ald-newborn-screening-result">Research Study: Families in California who received a positive ALD newborn screening result</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2018-11-01" itemprop="datePublished" pubdate><a href="/news-1/2018/11/1/families-in-california-who-received-a-positive-ald-newborn-screening-result">November 1, 2018</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>  <div class="special-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Promoted Block" data-type="promoted-block" id="promoted-block-5bdad323b8a0458599c6aedb"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block image-block sqs-block-image" data-aspect-ratio="29.428571428571427" data-block-type="5" id="block-yui_3_17_2_1_1541067533764_18202"><div class="sqs-block-content"> <div class=" image-block-outer-wrapper layout-caption-below design-layout-inline combination-animation-none individual-animation-none individual-text-animation-none " data-test="image-block-inline-outer-wrapper" > <figure class=" sqs-block-image-figure intrinsic " style="max-width:350px;" > <div class="image-block-wrapper" data-animation-role="image" > <div class="sqs-image-shape-container-element has-aspect-ratio " style=" position: relative; padding-bottom:29.428571701049805%; overflow: hidden;-webkit-mask-image: -webkit-radial-gradient(white, black); " > <img data-stretch="false" data-src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png" data-image="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png" data-image-dimensions="350x120" data-image-focal-point="0.5,0.5" alt="Exploring the impact of positive newborn screening results for X-linked adrenoleukodystrophy (X-ALD) among families in California" data-load="false" elementtiming="system-image-block" src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png" width="350" height="120" alt="" sizes="(max-width: 640px) 100vw, (max-width: 767px) 100vw, 100vw" style="display:block;object-fit: cover; width: 100%; height: 100%; object-position: 50% 50%" onload="this.classList.add("loaded")" srcset="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png?format=100w 100w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png?format=300w 300w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png?format=500w 500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png?format=750w 750w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png?format=1000w 1000w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png?format=1500w 1500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1541068131205-10QI8MQKIR014Y71F3ZC/california_state_u-stanislaus.png?format=2500w 2500w" loading="lazy" decoding="async" data-loader="sqs"> </div> </div> <figcaption class="image-caption-wrapper"> <div class="image-caption"><p><strong>Exploring the impact of positive newborn screening results for X-linked adrenoleukodystrophy (X-ALD) among families in California</strong></p></div> </figcaption> </figure> </div> </div></div></div></div></div></div>  <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1541068202662" id="item-5bdad323b8a0458599c6aedb"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-a1632eea7153417d0d97"><div class="sqs-block-content"> <div class="sqs-html-content"> <p style="white-space: pre-wrap;"><a href="https://www.linkedin.com/in/katharinaschwan/" target="_blank">Katharina Schwan</a>, who studies genetic counseling at California State University in the San Francisco/Bay Area, is conducting a research study on families who have received a positive ALD newborn screening result. Participants receive $25 compensation for their time. Please <a href="https://katschwan.wixsite.com/newbornscreeningxald" target="_blank">click here</a> for more information.</p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2018/11/1/families-in-california-who-received-a-positive-ald-newborn-screening-result#comments-5bdad323b8a0458599c6aedb">1 Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/5bdad323b8a0458599c6aedb/1541068294472/" data-record-type="1" data-full-url="/news-1/2018/11/1/families-in-california-who-received-a-positive-ald-newborn-screening-result" data-title="Research Study: Families in California who received a positive ALD newborn screening result"></span> <span class="sqs-simple-like" data-item-id="5bdad323b8a0458599c6aedb" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-10" id="article-5a4e5b92ec212d1b66b0e56b" data-item-id="5a4e5b92ec212d1b66b0e56b">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2018/1/4/100000-adrenomyeloneuropathy-amn-research-grant-awarded-to-ali-fatemi-md-at-the-kennedy-krieger-institute">$100,000 Adrenomyeloneuropathy (AMN) Research Grant Awarded to Ali Fatemi, MD at The Kennedy Krieger Institute</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2018-01-04" itemprop="datePublished" pubdate><a href="/news-1/2018/1/4/100000-adrenomyeloneuropathy-amn-research-grant-awarded-to-ali-fatemi-md-at-the-kennedy-krieger-institute">January 4, 2018</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1515085107687" id="item-5a4e5b92ec212d1b66b0e56b"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-b2358ce72cf3b18658ff"><div class="sqs-block-content"> <div class="sqs-html-content"> <p>The <a target="_blank" href="http://stopald.org">Stop ALD Foundation</a> is excited to announce that the <a target="_blank" href="https://www.med.upenn.edu/orphandisease/">University of Pennsylvania's Orphan Disease Center</a> has awarded a $100,000 research pilot grant to <a target="_blank" href="https://www.kennedykrieger.org/patient-care/faculty-staff/s-ali-fatemi">Dr. Ali Fatemi</a> at <a target="_blank" href="https://www.kennedykrieger.org/">The Kennedy Krieger Institute</a>.</p><p>A significant hurdle for translating preclinical work into clinical trials for <a target="_blank" href="http://www.stopald.org/what-is-ald/">Adrenomyeloneuropathy (AMN)</a> is the slowly progressive nature of AMN with extremely variable rates of progression. There is a critical need for surrogate biomarkers which can predict disease progression, or identify individuals who are rapid progressors. Effort is needed to be “clinical trial ready” so that therapeutic treatments can be tested for efficacy. </p><p>This grant will fund assessment of existing <a target="_blank" href="https://www.kennedykrieger.org/">Kennedy Krieger</a> longitudinal magnetic transfer (MT) and diffuse tensor imaging (DTI) magnetic resonance imaging (MRI) of the brain and cervical spinal cord of AMN patients to look for significant changes over a 12-month period. In addition, standardized neuroimaging protocols will be implemented which can be executed at 4 main <a target="_blank" href="http://aldconnect.org/">ALD Connect</a> clinical trial centers. The identification and implementation of a multicenter imaging protocol for AMN is needed to allow the full implementation of the ALD Connect multicenter clinical trial network.</p><p>This work will serve as an important foundation for the broad research community, supporting a range of therapeutic approaches being studied by many dedicated researchers. </p><p>This $100,000 grant was a result of the University of Pennsylvania Orphan Disease Center's annual <a target="_blank" href="http://www.milliondollarbikeride.org/">Million Dollar Bike Ride</a>. The Million Dollar Bike Ride focuses on raising money for rare disease research doubling the amount of money raised by its participants. The Stop ALD Foundation lead the ALD team and raised a total of $50,000, of which The Stop ALD Foundation contributed $40,000, amplifying donor money into important research areas. Dr. Fatemi’s grant will advance much-needed research.</p><p><strong>Grant Awarded: $100,000</strong></p><p><strong>Project Title</strong>: Multicenter Cross Standardization of Magnetic Resonance Imaging Methods for Adrenoleukodystrophy/Adrenomyeloneuropathy.</p><p><strong>Project Goal</strong>: Implement, cross standardize and validate MRI protocols necessary for clinical trials in Adrenomyeloneuropathy (AMN), the adult form of X-linked Adrenoleukodystrophy (ALD), for the ALD Connect Consortium.</p><p><strong>Brief Background</strong>: Adrenomyeloneuropathy (AMN) is the adult variant of X-linked ALD and is a slowly progressive neurodegenerative disorder due to mutations in the peroxisomal AcylCoA transporter, ABCD1, with no current therapeutic options. However, over the last 10 years, at least 5 different biotechnology companies have developed a research and development program for AMN and encouraging results are emerging from preclinical studies. Furthermore, several neuroimaging methods have been studied in AMN and could serve as biomarkers. Yet, the conduction of clinical trials is hampered by rarity of the disorder and lack of standardized quantitative outcome measures. In an effort to overcome these issues, ALD Connect, a new consortium, was established a few years ago with the goal to cross-harmonize data collection. </p><p>Importantly, <strong>MRI imaging protocols are not standardized between centers specialized in ALD for clinical trials, due to varying MRI scanner models and paradigms</strong>. In collaboration with Dr. Seth Smith, we have developed tools that will allow the harmonization of imaging protocols between the nation’s five main clinical sites.</p><p><strong>Specific Aims:</strong></p><ul dir="ltr"><li style="margin-left: 40px;"><strong>Aim 1:</strong> Develop a standardized imaging protocol and MRI sequence for AMN trials which is executable by all included site MRI scanners and infrastructure.</li><li style="margin-left: 40px;"><strong>Aim 2:</strong> Implement imaging protocols by performing on-location MRI brain scans of a single healthy control subject at each trial site. Assess equivalency of MRI scan data between sites at The Kennedy Krieger Institute, and implement necessary protocol changes at the trial sites. Establish a communication and review platform between AMN and imaging experts for the trial sites as a multi-center imaging network.</li></ul> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2018/1/4/100000-adrenomyeloneuropathy-amn-research-grant-awarded-to-ali-fatemi-md-at-the-kennedy-krieger-institute#comments-5a4e5b92ec212d1b66b0e56b">8 Comments</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/5a4e5b92ec212d1b66b0e56b/1515085107976/" data-record-type="1" data-full-url="/news-1/2018/1/4/100000-adrenomyeloneuropathy-amn-research-grant-awarded-to-ali-fatemi-md-at-the-kennedy-krieger-institute" data-title="$100,000 Adrenomyeloneuropathy (AMN) Research Grant Awarded to Ali Fatemi, MD at The Kennedy Krieger Institute"></span> <span class="sqs-simple-like" data-item-id="5a4e5b92ec212d1b66b0e56b" data-like-count="6"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry promoted promoted-block-image author-steve-barsh post-type-text article-index-11" id="article-59d6d769cd0f68e30c03a7da" data-item-id="59d6d769cd0f68e30c03a7da">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2017/10/5/ny-times-in-a-first-gene-therapy-halts-a-fatal-brain-disease">NY Times: In a First, Gene Therapy Halts a Fatal Brain Disease</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2017-10-05" itemprop="datePublished" pubdate><a href="/news-1/2017/10/5/ny-times-in-a-first-gene-therapy-halts-a-fatal-brain-disease">October 5, 2017</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>  <div class="special-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Promoted Block" data-type="promoted-block" id="promoted-block-59d6d769cd0f68e30c03a7da"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block image-block sqs-block-image" data-block-type="5" id="block-yui_3_17_2_1_1507252054308_18527"><div class="sqs-block-content"> <figure class=" sqs-block-image-figure image-block-outer-wrapper image-block-v2 design-layout-poster image-position-left " data-scrolled data-test="image-block-v2-outer-wrapper" > <div class="intrinsic"> <div class=" image-inset" data-animation-role="image" data-description="" data-animation-override > <div class="sqs-image-shape-container-element content-fill has-aspect-ratio " style=" position: relative; overflow: hidden;-webkit-mask-image: -webkit-radial-gradient(white, black); padding-bottom:66.650390625%; "> <img data-stretch="false" data-src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg" data-image="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg" data-image-dimensions="2048x1365" data-image-focal-point="0.5,0.5" alt="06GENES1-superJumbo.jpg" data-load="false" elementtiming="system-image-block" src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg" width="2048" height="1365" alt="" sizes="100vw" style="display:block;object-fit: cover; width: 100%; height: 100%; object-position: 50% 50%" onload="this.classList.add("loaded")" srcset="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg?format=100w 100w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg?format=300w 300w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg?format=500w 500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg?format=750w 750w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg?format=1000w 1000w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg?format=1500w 1500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507252129200-JV677VHANPL5689XGPFH/06GENES1-superJumbo.jpg?format=2500w 2500w" loading="lazy" decoding="async" data-loader="sqs"> <div class="image-overlay" style="overflow: hidden;-webkit-mask-image: -webkit-radial-gradient(white, black);"></div> </div> </div> </div> </figure> </div></div></div></div></div></div>  <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1507252217636" id="item-59d6d769cd0f68e30c03a7da"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-f58baa52b4f123f0b108"><div class="sqs-block-content"> <div class="sqs-html-content"> <h1>In a First, Gene Therapy Halts a Fatal Brain Disease</h1><p>For the first time, doctors have used gene therapy to stave off a fatal degenerative brain disease, an achievement that some experts had thought impossible.</p><p>The key to making the therapy work? One of the medicine’s greatest villains: HIV.</p><p>The patients were children who had inherited a mutated gene causing a rare disorder, <a href="http://www.stopald.org/what-is-ald/">adrenoleukodystrophy</a>, or ALD. Nerve cells in the brain die, and in a few short years, children lose the ability to walk or talk. <a target="_blank" href="https://www.nytimes.com/2017/10/05/health/gene-therapy-brain-disease.html?_r=0">More...</a></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2017/10/5/ny-times-in-a-first-gene-therapy-halts-a-fatal-brain-disease#comments-59d6d769cd0f68e30c03a7da">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/59d6d769cd0f68e30c03a7da/1507252217944/" data-record-type="1" data-full-url="/news-1/2017/10/5/ny-times-in-a-first-gene-therapy-halts-a-fatal-brain-disease" data-title="NY Times: In a First, Gene Therapy Halts a Fatal Brain Disease"></span> <span class="sqs-simple-like" data-item-id="59d6d769cd0f68e30c03a7da" data-like-count="2"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry promoted promoted-block-image author-steve-barsh post-type-text article-index-12" id="article-59d54dc1cd39c37f3ba513ae" data-item-id="59d54dc1cd39c37f3ba513ae">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2017/10/4/npr-parents-lobby-states-to-expand-newborn-screening-test-for-rare-brain-disorder">NPR: Parents Lobby States To Expand Newborn Screening Test For Rare Brain Disorder</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2017-10-04" itemprop="datePublished" pubdate><a href="/news-1/2017/10/4/npr-parents-lobby-states-to-expand-newborn-screening-test-for-rare-brain-disorder">October 4, 2017</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>  <div class="special-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Promoted Block" data-type="promoted-block" id="promoted-block-59d54dc1cd39c37f3ba513ae"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block image-block sqs-block-image" data-block-type="5" id="block-yui_3_17_2_1_1507151271851_18703"><div class="sqs-block-content"> <figure class=" sqs-block-image-figure image-block-outer-wrapper image-block-v2 design-layout-poster image-position-left " data-scrolled data-test="image-block-v2-outer-wrapper" > <div class="intrinsic"> <div class=" image-inset" data-animation-role="image" data-description="" data-animation-override > <div class="sqs-image-shape-container-element content-fill has-aspect-ratio " style=" position: relative; overflow: hidden;-webkit-mask-image: -webkit-radial-gradient(white, black); padding-bottom:74.875%; "> <img data-stretch="false" data-src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg" data-image="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg" data-image-dimensions="800x599" data-image-focal-point="0.5,0.5" alt="newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg" data-load="false" elementtiming="system-image-block" src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg" width="800" height="599" alt="" sizes="100vw" style="display:block;object-fit: cover; width: 100%; height: 100%; object-position: 50% 50%" onload="this.classList.add("loaded")" srcset="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg?format=100w 100w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg?format=300w 300w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg?format=500w 500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg?format=750w 750w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg?format=1000w 1000w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg?format=1500w 1500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1507151416797-58P4GO9FSL1CALJETJJB/newborn-screening-1-a35f72ad2e704149a5614c846f621c9fc08102aa-s800-c85.jpg?format=2500w 2500w" loading="lazy" decoding="async" data-loader="sqs"> <div class="image-overlay" style="overflow: hidden;-webkit-mask-image: -webkit-radial-gradient(white, black);"></div> </div> </div> </div> </figure> </div></div></div></div></div></div>  <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1507151544553" id="item-59d54dc1cd39c37f3ba513ae"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-48bd38aef84a7d6d53a8"><div class="sqs-block-content"> <div class="sqs-html-content"> <h1>Parents Lobby States To Expand Newborn Screening Test For Rare Brain Disorder</h1><p>Kerri De Nies received the news this spring from her son's pediatrician: Her chubby-cheeked toddler has a rare brain disorder.</p><p>She'd never heard of the disease — <a href="https://ghr.nlm.nih.gov/condition/x-linked-adrenoleukodystrophy">adrenoleukodystrophy</a>, or ALD — but soon felt devastated and overwhelmed.</p><p>"I probably read everything you could possibly read online — every single website," De Nies says as she cradles her son, Gregory Mac Phee.<strong> </strong>"It's definitely hard to think about what could potentially happen. You think about the worst-case scenario." <a target="_blank" href="http://www.npr.org/sections/health-shots/2017/10/04/555091418/parents-lobby-states-to-expand-newborn-screening-test-for-rare-brain-disorder"><em>More...</em></a></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2017/10/4/npr-parents-lobby-states-to-expand-newborn-screening-test-for-rare-brain-disorder#comments-59d54dc1cd39c37f3ba513ae">2 Comments</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/59d54dc1cd39c37f3ba513ae/1507151544997/" data-record-type="1" data-full-url="/news-1/2017/10/4/npr-parents-lobby-states-to-expand-newborn-screening-test-for-rare-brain-disorder" data-title="NPR: Parents Lobby States To Expand Newborn Screening Test For Rare Brain Disorder"></span> <span class="sqs-simple-like" data-item-id="59d54dc1cd39c37f3ba513ae" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-13" id="article-59a22fc68419c2bb441276df" data-item-id="59a22fc68419c2bb441276df">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2017/8/26/2017-million-dollar-bike-ride-pilot-grant-program-request-for-applications">2017 Million Dollar Bike Ride Pilot Grant Program: Request for Applications</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2017-08-26" itemprop="datePublished" pubdate><a href="/news-1/2017/8/26/2017-million-dollar-bike-ride-pilot-grant-program-request-for-applications">August 26, 2017</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1503801342965" id="item-59a22fc68419c2bb441276df"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-6539ba841f724f5cd6f3"><div class="sqs-block-content"> <div class="sqs-html-content"> <p>The 2017 Million Dollar Bike Ride Pilot Grant Program is now open!  The MDBR Pilot Grant Program provides a one-year grant to support research related to a rare disease represented in the 2017 Million Dollar Bike Ride. Thirty-three (33) total grant opportunities are listed below.</p><p><strong>Eligibility: </strong>All individuals holding a faculty-level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA.  </p><p><strong>To Apply:</strong>  Please review the RFA guidelines <a href="http://www.med.upenn.edu/orphandisease/assets/user-content/documents/2017%20MDBR%20Pilot%20Grant%20RFA.pdf">here. </a> Interested applicants <strong>must first submit a Letter of Interest</strong> (LOI) which can be found <a target="_blank" href="https://somapps.med.upenn.edu/apps/form/orphandisease/view.php?id=135366">here</a>.  <strong>This LOI is due by Monday, September 18, 2017 by 8pm EST.   Full applications are accepted by <em>invitation only</em> after LOIs are approved. </strong></p><p><strong>Research Focus Areas for Pilot Grants:</strong></p><p><strong>1) </strong><strong>Adrenoleukodystrophy (ALD):</strong> One $101,164 pilot grant is available with a focus on a path towards treatment for Adrenomyeloneuropathy (AMN). Grants should focus on activities that lead towards a clinical trial.  Adrenoleukodystrophy (ALD) is an X-linked metabolic disorder, characterized by progressive neurologic deterioration due to demyelination of the cerebral white matter. The adult form, Adrenomyeloneuropathy (AMN), develops in young adulthood and in general, progresses more slowly than ALD. Beginning in their 20s and 30s, men and ~60% of women carriers exhibit neurological-based motor lesions in their extremities. These lesions progress over many years and are characterized by progressive spasticity, ataxia, incontinence, and sexual dysfunction that can also be accompanied by fatigue and depression. ALD/AMN can lead to severe disability. All current treatments are symptomatic and do not address the progressive nature of the disease. This grant is made possible by Team Stop ALD and the Stop ALD Foundation.</p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2017/8/26/2017-million-dollar-bike-ride-pilot-grant-program-request-for-applications#comments-59a22fc68419c2bb441276df">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/59a22fc68419c2bb441276df/1503801405565/" data-record-type="1" data-full-url="/news-1/2017/8/26/2017-million-dollar-bike-ride-pilot-grant-program-request-for-applications" data-title="2017 Million Dollar Bike Ride Pilot Grant Program: Request for Applications"></span> <span class="sqs-simple-like" data-item-id="59a22fc68419c2bb441276df" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-14" id="article-5897e376b3db2ba7b8d8942c" data-item-id="5897e376b3db2ba7b8d8942c">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2017/2/5/medday-md1003-clinical-trial-data">MedDay MD1003 clinical trial data</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2017-02-05" itemprop="datePublished" pubdate><a href="/news-1/2017/2/5/medday-md1003-clinical-trial-data">February 5, 2017</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1486349334933" id="item-5897e376b3db2ba7b8d8942c"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-fa5bb84e44f975ce9759"><div class="sqs-block-content"> <div class="sqs-html-content"> <p><span style="font-size:13px">Per <a target="_blank" href="http://www.medday-pharma.com/">MedDay</a>, the final <a target="_blank" href="http://www.medday-pharma.com/research-development/pipeline/md1003/">MD1003</a> clinical trial data in AMN patients is expected the end of 2017.</span></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2017/2/5/medday-md1003-clinical-trial-data#comments-5897e376b3db2ba7b8d8942c">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/5897e376b3db2ba7b8d8942c/1486349335001/" data-record-type="1" data-full-url="/news-1/2017/2/5/medday-md1003-clinical-trial-data" data-title="MedDay MD1003 clinical trial data"></span> <span class="sqs-simple-like" data-item-id="5897e376b3db2ba7b8d8942c" data-like-count="3"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-15" id="article-585fd493d2b857ddbd6e89f3" data-item-id="585fd493d2b857ddbd6e89f3">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2016/12/25/100000-amn-research-grant-awarded-to-dr-florian-eichler-massachusetts-general-hospital">$100,000 AMN Research Grant Awarded to Dr. Florian Eichler, Massachusetts General Hospital</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2017-01-03" itemprop="datePublished" pubdate><a href="/news-1/2016/12/25/100000-amn-research-grant-awarded-to-dr-florian-eichler-massachusetts-general-hospital">January 3, 2017</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1482676704744" id="item-585fd493d2b857ddbd6e89f3"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-7b892dc13152f8d89e1c"><div class="sqs-block-content"> <div class="sqs-html-content"> <p>The Stop ALD Foundation is excited to announce that the <a target="_blank" href="https://www.med.upenn.edu/orphandisease/">University of Pennsylvania's Orphan Disease Center</a> has awarded a one-year, $100,000 research pilot grant to <a target="_blank" href="http://www.massgeneral.org/children/doctors/doctor.aspx?id=17851">Dr. Florian Eichler</a> at Massachusetts General Hospital. Dr. Eichler and his team have already established an Adrenomyeloneuropathy (AMN) animal model proof of concept showing a gene therapy approach could provide benefit where no treatment is currently available. Today when young men with Adrenoleukodystrophy (ALD) begin showing adult-onset signs of AMN, they slowly lose their ability to walk. This pilot grant will help move Dr. Eichler's research program forward towards human studies. However, additional funds will still be necessary to initiate the study.</p><p>This $100,000 grant was a result of the Penn Orphan Disease Center's annual <a target="_blank" href="http://www.milliondollarbikeride.org/">Million Dollar Bike Ride</a>. The Million Dollar Bike ride <a target="_blank" href="http://www.milliondollarbikeride.org/about-us/">focuses on raising money</a> for rare disease research doubling money raised. The Stop ALD Foundation lead the ALD Team and raised a total of $50,000 of which The Stop ALD Foundation contributed $40,000, amplifying donor money into important research areas. Dr. Eichler's grant will advance much-needed research into a promising treatment for Adrenomyeloneuropathy (AMN). This $100,000 pilot grant is just the beginning and we encourage other donors and foundations to fund this important AMN research further to help it move towards a clinical trial.</p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2016/12/25/100000-amn-research-grant-awarded-to-dr-florian-eichler-massachusetts-general-hospital#comments-585fd493d2b857ddbd6e89f3">10 Comments</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/585fd493d2b857ddbd6e89f3/1483455206263/" data-record-type="1" data-full-url="/news-1/2016/12/25/100000-amn-research-grant-awarded-to-dr-florian-eichler-massachusetts-general-hospital" data-title="$100,000 AMN Research Grant Awarded to Dr. Florian Eichler, Massachusetts General Hospital"></span> <span class="sqs-simple-like" data-item-id="585fd493d2b857ddbd6e89f3" data-like-count="16"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-16" id="article-57d62f2a197aea51693773a4" data-item-id="57d62f2a197aea51693773a4">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2016/9/12/100000-aldamn-research-grant-application-due-sept-23-2016">$100,000 ALD/AMN Research Grant. Application Due Sept 23, 2016</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2016-09-12" itemprop="datePublished" pubdate><a href="/news-1/2016/9/12/100000-aldamn-research-grant-application-due-sept-23-2016">September 12, 2016</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1473654733302" id="item-57d62f2a197aea51693773a4"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-b894cea05a16ccbd11fc"><div class="sqs-block-content"> <div class="sqs-html-content"> <h3>2016 Million Dollar Bike Ride Grants</h3><p>The 2016 Million Dollar Bike Ride Pilot Grant Program is now open!  The MDBR Pilot Grant Program provides a one-year grant to support research related ALD/AMN. </p><p><strong>Eligibility: </strong>All individuals holding a faculty-level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA.  </p><p><strong>To Apply: </strong>Please be sure to read the <a href="http://www.med.upenn.edu/orphandisease/assets/user-content/documents/2016%20MDBR%20RFA%20Guidelines.pdf">MDBR RFA Guidelines </a>before applying.  Submit your Letter of Interest (LOI) via the link below no later than <strong>Friday, September 23, 2016 by 8pm EST. </strong>Full application is by invitation only, after review of LOIs. </p><p><strong>LOI Submission Form:</strong> <a target="_blank" href="https://somapps.med.upenn.edu/apps/form/orphandisease/view.php?id=108815">LOI Submission Form </a> <strong>Due 9/23/16 by 8pm EST. </strong></p><p><strong>Research Focus Areas for Pilot Grant:</strong></p><p><strong>Adrenoleukodystrophy (ALD):</strong> One $100,000 pilot grant is available with a focus on a path towards treatment for Adrenomyeloneuropathy (AMN). Adrenoleukodystrophy (ALD) is an x-linked metabolic disorder, characterized by progressive neurologic deterioration due to demyelination of the cerebral white matter. The adult form, Adrenomyeloneuropathy (AMN), develops in young adulthood and in general, progresses more slowly than ALD. Beginning in their 20s and 30s, men and ~60% of women carriers exhibit neurological-based motor lesions in their extremities. These lesions progress over many years and are characterized by progressive spasticity, ataxia, incontinence, and sexual dysfunction that can also be accompanied by fatigue and depression. ALD/AMN can lead to severe disability. All current treatments are symptomatic and do not address the progressive nature of the disease. This grant is made possible by Team Stop ALD and the Stop ALD Foundation.</p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2016/9/12/100000-aldamn-research-grant-application-due-sept-23-2016#comments-57d62f2a197aea51693773a4">2 Comments</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/57d62f2a197aea51693773a4/1473654733563/" data-record-type="1" data-full-url="/news-1/2016/9/12/100000-aldamn-research-grant-application-due-sept-23-2016" data-title="$100,000 ALD/AMN Research Grant. Application Due Sept 23, 2016"></span> <span class="sqs-simple-like" data-item-id="57d62f2a197aea51693773a4" data-like-count="1"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-17" id="article-572e3cfe7da24fce8269d9c9" data-item-id="572e3cfe7da24fce8269d9c9">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2016/5/7/great-ride-at-upenn-million-dollar-bike-ride-for-rare-diseases">Great ride at UPenn Million Dollar Bike Ride for Rare Diseases</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2016-05-07" itemprop="datePublished" pubdate><a href="/news-1/2016/5/7/great-ride-at-upenn-million-dollar-bike-ride-for-rare-diseases">May 7, 2016</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1462648283545" id="item-572e3cfe7da24fce8269d9c9"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-2f7a72d131305702646d"><div class="sqs-block-content"> <div class="sqs-html-content"> <p>Cold and rainy and worth every minute of it!  The Stop ALD Foundation participated in the annual Million Dollar Bike Ride at UPenn in Philly on May 7, 2016. We have until May 30, 2016 to accept donations. Our goal is $20k and at that goal our fundraising will be doubled to $40k.  <a target="_blank" href="https://socialfundraising.apps.upenn.edu/socialFundraising/jsp/fast.do?fastStart=customTemplate&id=100955503&utm_content=buffer19c6f&utm_medium=social&utm_source=twitter.com&utm_campaign=buffer">Click here to make a donation</a>. Honored to ride today with <a target="_blank" href="http://www.curefa.org/index.php">Kyle Bryant of FARA</a>!</p> </div> </div></div><div class="sqs-block image-block sqs-block-image" data-block-type="5" id="block-yui_3_17_2_1_1462648043838_20680"><div class="sqs-block-content"> <div class=" image-block-outer-wrapper layout-caption-below design-layout-inline " data-test="image-block-inline-outer-wrapper" > <figure class=" sqs-block-image-figure intrinsic " style="max-width:640px;" > <div class="image-block-wrapper" data-animation-role="image" data-animation-override > <div class="sqs-image-shape-container-element has-aspect-ratio " style=" position: relative; padding-bottom:75%; overflow: hidden;-webkit-mask-image: -webkit-radial-gradient(white, black); " > <img data-stretch="false" data-src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg" data-image="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg" data-image-dimensions="640x480" data-image-focal-point="0.5,0.5" alt="" data-load="false" elementtiming="system-image-block" src="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg" width="640" height="480" alt="" sizes="(max-width: 640px) 100vw, (max-width: 767px) 100vw, 100vw" style="display:block;object-fit: cover; width: 100%; height: 100%; object-position: 50% 50%" onload="this.classList.add("loaded")" srcset="https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg?format=100w 100w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg?format=300w 300w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg?format=500w 500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg?format=750w 750w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg?format=1000w 1000w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg?format=1500w 1500w, https://images.squarespace-cdn.com/content/v1/52e89bfae4b06b90fc8aa357/1462648214584-T6WZM7YT15AWMZOFJEXI/image-asset.jpeg?format=2500w 2500w" loading="lazy" decoding="async" data-loader="sqs"> </div> </div> </figure> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2016/5/7/great-ride-at-upenn-million-dollar-bike-ride-for-rare-diseases#comments-572e3cfe7da24fce8269d9c9">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/572e3cfe7da24fce8269d9c9/1462648284252/" data-record-type="1" data-full-url="/news-1/2016/5/7/great-ride-at-upenn-million-dollar-bike-ride-for-rare-diseases" data-title="Great ride at UPenn Million Dollar Bike Ride for Rare Diseases"></span> <span class="sqs-simple-like" data-item-id="572e3cfe7da24fce8269d9c9" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-18" id="article-56fb65927da24fbb66367dc2" data-item-id="56fb65927da24fbb66367dc2">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2016/3/30/philly-3rd-annual-million-dollar-bike-ride-for-rare-diseases">Philly 3rd Annual Million Dollar Bike Ride for Rare Diseases</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2016-03-30" itemprop="datePublished" pubdate><a href="/news-1/2016/3/30/philly-3rd-annual-million-dollar-bike-ride-for-rare-diseases">March 30, 2016</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1459316147526" id="item-56fb65927da24fbb66367dc2"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-25cee60288a403c99dba"><div class="sqs-block-content"> <div class="sqs-html-content"> <p><span style="font-size:14px">The 3rd Annual Million Dollar Bike Ride for Rare Diseases is coming up May 7th in Philadelhphia. Please consider riding or making a donation where UPenn has a grant to DOUBLE all donations. Great chance to make a difference and have your contribution doubled! </span><a target="_blank" href="http://www.milliondollarbikeride.org/">http://www.milliondollarbikeride.org/</a></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2016/3/30/philly-3rd-annual-million-dollar-bike-ride-for-rare-diseases#comments-56fb65927da24fbb66367dc2">Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/56fb65927da24fbb66367dc2/1459316147725/" data-record-type="1" data-full-url="/news-1/2016/3/30/philly-3rd-annual-million-dollar-bike-ride-for-rare-diseases" data-title="Philly 3rd Annual Million Dollar Bike Ride for Rare Diseases"></span> <span class="sqs-simple-like" data-item-id="56fb65927da24fbb66367dc2" data-like-count="0"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-19" id="article-56c8a56b8a65e2903b66d726" data-item-id="56c8a56b8a65e2903b66d726">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2016/2/20/secretary-of-health-and-human-services-accepts-the-recommendation-to-expand-the-recommended-uniform-screening-panel-to-include-x-ald-and-mps-i">Secretary of Health and Human Services accepts the recommendation to expand the Recommended Uniform Screening Panel to include X-ALD and MPS I</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2016-02-20" itemprop="datePublished" pubdate><a href="/news-1/2016/2/20/secretary-of-health-and-human-services-accepts-the-recommendation-to-expand-the-recommended-uniform-screening-panel-to-include-x-ald-and-mps-i">February 20, 2016</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1455990258208" id="item-56c8a56b8a65e2903b66d726"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-9ca0476b1d5a12f3efe3"><div class="sqs-block-content"> <div class="sqs-html-content"> <p>Secretary of Health and Human Services Sylvia Mathews Burwell, accepted the recommendation to expand the Recommended Uniform Screening Panel (RUSP) to include the addition of X-linked Adrenoleukodystrophy (X-ALD) and Mucopolysaccharidosis type 1 (MPS I). The Affordable Care Act requires that most health plans cover evidence-based preventative care and screenings provided for in the comprehensive guidelines supported by Health Resources and Service Administration (HRSA).</p><p><a target="_blank" href="http://www.webwire.com/ViewPressRel.asp?aId=202180">More... </a></p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2016/2/20/secretary-of-health-and-human-services-accepts-the-recommendation-to-expand-the-recommended-uniform-screening-panel-to-include-x-ald-and-mps-i#comments-56c8a56b8a65e2903b66d726">1 Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/56c8a56b8a65e2903b66d726/1455990258272/" data-record-type="1" data-full-url="/news-1/2016/2/20/secretary-of-health-and-human-services-accepts-the-recommendation-to-expand-the-recommended-uniform-screening-panel-to-include-x-ald-and-mps-i" data-title="Secretary of Health and Human Services accepts the recommendation to expand the Recommended Uniform Screening Panel to include X-ALD and MPS I"></span> <span class="sqs-simple-like" data-item-id="56c8a56b8a65e2903b66d726" data-like-count="2"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article> <article class="hentry author-steve-barsh post-type-text article-index-20" id="article-55e1a54fe4b05355474e4c8b" data-item-id="55e1a54fe4b05355474e4c8b">  <header> <h1 class="entry-title" itemprop="headline"> <a href="/news-1/2015/8/29/rfa-for-50000-for-amn-research">RFA for $50,000 for AMN research</a> </h1> <div class="meta"> <span class="date"><time class="published dt-published" datetime="2015-08-29" itemprop="datePublished" pubdate><a href="/news-1/2015/8/29/rfa-for-50000-for-amn-research">August 29, 2015</a></time></span> <span class="author"><a href="/news-1?author=52e89bf9e4b00b97daffaafb" rel="author">Steve Barsh</a></span> </div> </header>   <div class="body entry-content"><div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Post Body" data-type="item" data-updated-on="1440851426533" id="item-55e1a54fe4b05355474e4c8b"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-4ee6ceaa5ec06f5bd52b"><div class="sqs-block-content"> <div class="sqs-html-content"> <p>The 2015 Million Dollar Bike Ride Pilot Grant Program is now open.  Please first review the RFA guidelines before submitting your pre-application using the webform below.  All pre-application submissions are due <strong>Monday, September 14, 2015 by 5:00 pm (EST).</strong>  If your pre-application is approved, you will be notified with an invitation to submit a full-application, due October 19, 2015.</p><p>For questions regarding this pilot grant program, please contact Samantha Charleston at <a href="mailto:scharle@mail.med.upenn.edu">scharle@mail.med.upenn.edu</a>, or (215) 573-6822.</p><p>Click here to view the 2015 MDBR Pilot Grant RFA Guidelines: <a target="_blank" href="http://www.med.upenn.edu/orphandisease/documents/2015MDBRRFAGuidelines.pdf">2015 MDBR Pilot Grant RFA Guidelines</a></p><p>Click here to submit your pre-application: </p><p><strong>Research Focus Areas for Pilot Grants:</strong></p><p><strong>Adrenoleukodystrophy (ALD):</strong> Adrenoleukodystrophy, or ALD, is an x-linked metabolic disorder, characterized by progressive neurologic deterioration due to demyelination of the cerebral white matter. The adult form, known as Adrenomyeloneuropathy (AMN) develops in young adulthood, and in general, they progress more slowly. Beginning in their 20s and 30s, these young men exhibit neurological based motor lesions in their extremities. These lesions progress over many years and are inevitably accompanied by moderate to severe handicap. In approximately one third of these patients the central nervous system also becomes involved.</p><p>One $50,000 pilot grant is available with a focus on treatments for Adrenomyeloneuropathy (AMN), the adult form of the disease. We are interested in proposals that would provide a path towards a treatment, including advancing the understanding of what can be used as endpoints when conducting an AMN trial. This grant is made possible by Team Stop ALD and the Stop ALD Foundation.</p> </div> </div></div></div></div></div></div>  <footer class="clear"> <div class="meta"> <div class="second meta-row"> <span class="comments"><a href="/news-1/2015/8/29/rfa-for-50000-for-amn-research#comments-55e1a54fe4b05355474e4c8b">1 Comment</a></span> <span class="squarespace-social-buttons inline-style" data-system-data-id="" data-asset-url="https://static1.squarespace.com/static/52e89bfae4b06b90fc8aa357/52eb3f6ae4b058d7164a4ae9/55e1a54fe4b05355474e4c8b/1440851426994/" data-record-type="1" data-full-url="/news-1/2015/8/29/rfa-for-50000-for-amn-research" data-title="RFA for $50,000 for AMN research"></span> <span class="sqs-simple-like" data-item-id="55e1a54fe4b05355474e4c8b" data-like-count="1"> <span class="like-icon"></span> <span class="like-count"></span> </span> </div> </div> </footer> </article>  <nav class="pagination clear"> <a class="older-posts" href="/news-1?offset=1440851426525">Older Posts →</a> </nav> </div> <aside id="sidebar"><div class="sqs-layout sqs-grid-1 columns-1 empty" data-layout-label="Sidebar Content" data-type="block-field" id="sidebarBlocks"><div class="row sqs-row"><div class="col sqs-col-1 span-1"></div></div></div></aside> </section> <div class="sqs-layout sqs-grid-12 columns-12 empty" data-layout-label="News Footer Content" data-type="block-field" id="collection-52eb3f6ae4b058d7164a4ae9"><div class="row sqs-row"><div class="col sqs-col-12 span-12"></div></div></div>  <div class="info-footer-wrapper clear"> <div class="info-footer"> <div class="sqs-layout sqs-grid-12 columns-12 empty" data-layout-label="Info Footer Content" data-type="block-field" data-updated-on="1395639618818" id="infoFooterBlock"><div class="row sqs-row"><div class="col sqs-col-12 span-12"></div></div></div> <div id="socialLinks" class="social-links sqs-svg-icon--list" data-content-field="connected-accounts"> <a href="http://www.facebook.com/stopald" target="_blank" class="sqs-svg-icon--wrapper facebook"> <div> <svg class="sqs-svg-icon--social" viewBox="0 0 64 64"> <use class="sqs-use--icon" xlink:href="#facebook-icon"></use> <use class="sqs-use--mask" xlink:href="#facebook-mask"></use> </svg> </div> </a> <a href="https://twitter.com/StopALD" target="_blank" class="sqs-svg-icon--wrapper twitter"> <div> <svg class="sqs-svg-icon--social" viewBox="0 0 64 64"> <use class="sqs-use--icon" xlink:href="#twitter-icon"></use> <use class="sqs-use--mask" xlink:href="#twitter-mask"></use> </svg> </div> </a> </div> </div> </div> <footer id="footer" class="clear"> <div class="sqs-layout sqs-grid-12 columns-12" data-layout-label="Footer Content" data-type="block-field" data-updated-on="1395639683170" id="footerBlock"><div class="row sqs-row"><div class="col sqs-col-12 span-12"><div class="sqs-block html-block sqs-block-html" data-block-type="2" id="block-7d9d7fddc71929b3cf64"><div class="sqs-block-content"> <div class="sqs-html-content"> <p>Copyright (c) 2004 - Present by The Stop ALD Foundation. 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