Testing BBM-D101 gene therapy for Duchenne muscular dystrophy
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This early-phase study tests a new gene therapy called BBM-D101 for boys with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and helps improve muscle function in young boys.
Summary written for real people, not researchers, by Clin2.
Who can take part
- The child must be a boy between 4 and 7 years old.
- A genetic test must confirm the child has Duchenne muscular dystrophy (DMD).
- The child must show at least one typical DMD symptom, like muscle weakness or a waddling walk.
- The child must be able to cooperate with tests like muscle MRI and a muscle biopsy.
- The child cannot have serious heart problems, active hepatitis B/C, HIV, or certain liver issues.
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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