Gene therapy for ambulant males with Duchenne muscular dystrophy
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new gene therapy called SGT-003 in boys and men with Duchenne muscular dystrophy who are still able to walk. The treatment aims to deliver a working version of the dystrophin gene to help muscle cells produce the protein they are missing.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are able to walk and run without help
- You have a confirmed genetic diagnosis of Duchenne muscular dystrophy
- You have not been treated with gene therapy before
- You have not taken certain other Duchenne medicines (like eteplirsen, golodirsen, ataluren, or vamorolone) within the past 6 months
- You weigh 50 kilograms (about 110 pounds) or less
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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