Gene therapy for duchenne muscular dystrophy
This trial tests a gene therapy to help boys with Duchenne muscular dystrophy (DMD) keep their muscle strength. It aims to see if the treatment is safe and effective for young boys who are still walking.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are a boy between 6 and 10 years old.
- You are able to walk and have a certain level of strength (NSAA score of at least 18).
- You have been taking steroids for at least 6 months before the study starts.
- You need to have regular vaccinations.
- You must not have certain genetic changes in the DMD gene (exons 1-17 or exons 8/9, depending on the study part).
- You must not have heart problems, breathing support, or antibodies to the virus used in the treatment.
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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