Experimental gene therapy shows promise for rare form of duchenne muscular dystrophy
NCT ID NCT04240314
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 22 times
Summary
This early-stage trial tested a gene therapy called scAAV9.U7.ACCA in 3 boys with Duchenne muscular dystrophy caused by a duplication of exon 2. The therapy was given as a single injection into a vein. The main goals were to check for serious side effects and to see if the treatment could increase dystrophin, a key muscle protein missing in Duchenne. The study is now complete.
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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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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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