Gene therapy study for Duchenne muscular dystrophy in boys 4-8
Part of Bones, joints & muscles, Genetic & congenital clinical trials.
This study tests a new gene therapy called BBM-D101 for boys with Duchenne muscular dystrophy. The goal is to see if it is safe and can help improve muscle function. It is for boys aged 4 to 8 who can still walk.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Boys aged 4 to under 9 years who can walk.
- A confirmed DMD gene mutation (genetic testing needed).
- Stable steroid use (e.g., prednisone) for a while before the study.
- No prior gene or cell therapy.
- Good overall health, with no serious heart, lung, liver, or immune problems.
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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