Gene therapy trial for Duchenne muscular dystrophy in young boys
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a gene therapy called GNR-097 for boys with Duchenne muscular dystrophy (DMD). The therapy aims to help improve muscle function, and the study will check how safe and effective it is.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Boys aged 4 to 9 who can walk and have a confirmed DMD diagnosis
- A specific type of DNA change (frameshift or nonsense mutation) in the DMD gene
- Must be taking a stable dose of oral steroids for at least 12 weeks
- Meningitis vaccine received at least 4 weeks before the study treatment
- Able to complete standard physical tests like standing up quickly and walking a certain distance
- No prior gene therapy or certain other medications
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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