Gene therapy for babies with severe spinal muscular atrophy
Part of Brain & nervous system clinical trials.
This early-stage study tests whether a one-time gene therapy can safely improve outcomes for babies with the most severe type of spinal muscular atrophy (SMA type 1). It focuses on safety and early signs of benefit, and participation requires specific genetic and health criteria.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be age 6 months or younger when the gene is given
- Have confirmed SMA type 1 based on genetic testing (two SMN1 gene changes and two SMN2 copies)
- Disease started before 6 months of age
- A parent/guardian must understand the study and sign informed consent
- Not have had earlier gene-therapy research treatment
- Do not have major exclusion conditions like certain immune markers, serious breathing needs, or active infection
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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