Gene therapy for children with limb-girdle muscular dystrophy
This study tests a new gene therapy called BBM-L101 for children with a specific type of muscular dystrophy (LGMD2D/R3). The hope is that the therapy can help replace a missing protein and improve muscle strength.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be between 4 and 13 years old
- Must have a confirmed genetic diagnosis of LGMD2D/R3
- Must have weak or absent alpha-sarcoglycan protein in a muscle biopsy
- Must have high CK levels (above 1000 U/L) and typical symptoms
- Must be on a stable dose of corticosteroids for at least 3 months
- Must be able to climb 4 stairs without help and complete required tests
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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