Gene therapy trial for spinal muscular atrophy type 3
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial is testing a one-time gene therapy drug for people with SMA type 3. The goal is to see if it can safely improve muscle function and strength.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 2 years old or older
- You have been diagnosed with SMA type 3 and genetic testing confirms the SMN1 gene deletion
- Your HFMSE score (a test of motor function) is between 10 and 54 at screening
- You have not had gene therapy before and have not used certain SMA medications (Nusinersen, Risdiplam) recently
- You do not need high levels of breathing support (16+ hours/day) or tube feeding, and you have no serious infections or allergies to prednisolone
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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