Gene therapy SGT-003 for 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 for children with Duchenne muscular dystrophy. It aims to help the body produce a working version of dystrophin, the protein missing in Duchenne.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be between 0 and 17 years old (different age groups available).
- Must be able to walk 10 meters in under 30 seconds (some groups allow wheelchair use if they used to walk).
- Must have a confirmed Duchenne diagnosis with a specific gene mutation.
- Must not have antibodies against AAV (a common virus used in gene therapy).
- For ages 4 and older: must be on a stable steroid treatment for at least 12 weeks.
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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