Gene therapy for type 2 spinal muscular atrophy
Part of Brain & nervous system clinical trials.
This trial tests a new gene therapy called GC101 given as a one-time injection into the spinal fluid for people with type 2 spinal muscular atrophy (SMA). It may help improve motor function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of type 2 SMA caused by changes in the SMN1 gene.
- You have been receiving Spinraza (nusinersen) treatment regularly for at least one year.
- You have not taken Evrysdi (risdiplam) in the last 2 months and can avoid it for 12 months after starting the trial.
- You can sit on your own but cannot walk independently, and your motor function score (HFMSE) is 10 or higher.
- You and your family are willing to follow the study rules and sign a consent form.
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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This study tests SKG0201, a new gene therapy designed to treat spinal muscular atrophy type I (SMA type I), a rare genetic condition affecting muscle strength. The trial enrolls very young infants diagnosed with SMA type I to see if the treatment is safe and helps improve muscle function.
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