New hope for duchenne kids: drug trial targets muscle damage after gene therapy
NCT ID NCT06100887
First seen Nov 06, 2025 · Last updated May 14, 2026 · Updated 20 times
Summary
This study tests an experimental drug called EDG-5506 (sevasemten) in 43 children aged 6 to 17 with Duchenne muscular dystrophy who have already received gene therapy. The goal is to see if the drug is safe and how it affects muscle health markers. Participants will first receive either the drug or a placebo, then everyone gets the drug in an open-label phase.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Rare Disease Research
Hillsborough, North Carolina, 27278, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01605, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
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