Duchenne Muscular Dystrophy
11 videos · last 6 months shuffled daily, then older videos newest first
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1:01:50 Deramiocel FDA Advisory Committee Town Hall
Parent Project Muscular Dystrophy and other Duchenne groups review the July 29, 2026 FDA advisory committee meeting on deramiocel, the committee's vote and what it means, and the steps leading to the FDA's August 22, 2026 action date.
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1:56 FDA Approval of a Duchenne Gene Therapy Developed at Nationwide
Nationwide Children's Hospital reports FDA approval of a gene therapy for Duchenne muscular dystrophy developed there, with principal investigator Jerry Mendell, MD, describing the single-infusion clinical trial in boys with DMD.
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23:11 Exon Skipping in Duchenne Muscular Dystrophy
Professor Francesco Muntoni reviews what is known so far from clinical trials about exon-skipping treatments for Duchenne muscular dystrophy, for Duchenne UK.
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4:40 Charles River Lab Works with Duchenne Muscular Dystrophy Disruptor
Duchenne muscular dystrophy ( DMD ), a fatal disease due to a mutation in a structural gene, impacted three generations of Rich Horgan's family and Rich used this experience to create Cure Rare Disease.
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6:55 Finding Clinical Trials for Muscular Dystrophy With ClinicalTrials.gov
In this video Hawken Miller explains how to use clinicaltrials.gov to find studies for Duchenne Muscular Dystrophy and looks at how to read a clinical trial result from completed studies.
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31:10 The Remote Collection of Multi-Modal Data for Decentralized Clinical Trials
Aparito's Dr Clare Matthews and Sandra Komarzynski present remote multi-modal data collection in decentralised trials, including the DMDHome app built with Duchenne UK to assess Duchenne patients at home through filmed tasks and questionnaires.
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19:20 The Story of Sarepta's Duchenne Gene Therapy Elevidys
STAT traces the development, approval and safety controversy of Sarepta's Duchenne gene therapy Elevidys and its back-and-forth with the FDA, with senior biotech writer Adam Feuerstein.
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16:34 How the DMD Hub Changed Access to Duchenne Trials
Emma Heslop describes how the DMD Hub has changed access to and participation in clinical research for Duchenne muscular dystrophy, at Duchenne UK's New Horizons conference.
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38:05 EXPLORE44 Topline Data for Del-Zota in Duchenne
Avidity Biosciences presents topline data from its EXPLORE44 trial of the investigational therapy delpacibart zotadirsen (del-zota) in people with Duchenne muscular dystrophy amenable to exon 44 skipping, in a Jett Foundation community webinar.
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25:51 Measuring Clinical Success in Duchenne Trials
Professor Laurent Servais discusses how clinical success is measured in Duchenne muscular dystrophy clinical trials, for Duchenne UK.
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16:02 Solid Bio and Parents Group Update on Duchenne Clinical Trial
A video from Solid Biosciences and PPMD with an update on the INSPIRE DUCHENNE clinical trial for SGT-003, a next-generation microdystrophin gene therapy for individuals living with Duchenne.