Gene therapy for children with type 2 SMA
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new gene therapy called GCB-001 for children with type 2 spinal muscular atrophy who can sit but not walk. It aims to improve muscle function and overall health.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 2 to 12 years
- Diagnosed with type 2 SMA (onset between 6 and 18 months, SMN1 gene mutation, 2-4 copies of SMN2 gene)
- Can sit without support but has never walked on their own
- No prior gene therapy or participation in other SMA drug trials
- No need for non-invasive ventilation for 12+ hours per day
- No serious health conditions (like heart, liver, kidney problems, epilepsy, or allergies to certain 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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