Study of salanersen for babies with SMA previously treated with gene therapy
Treatments studied
Part of Brain & nervous system clinical trials.
This study tests a new medicine called salanersen in babies who have already received gene therapy for SMA. It aims to see if adding salanersen is safe and effective.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a genetic diagnosis of SMA type 1 (with 2 copies of SMN2 gene).
- You received gene therapy (onasemnogene abeparvovec) before 42 days old and started screening within 6 months of that dose.
- You had no symptoms of SMA at the time of gene therapy (like normal reflexes and muscle responses).
- You have no ongoing abnormal lab results from gene therapy, such as high liver enzymes or low platelets.
- You haven't used any other SMA medicines besides gene therapy.
- You haven't used steroids related to gene therapy complications in the last 14 days.
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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