Long-term follow-up after EXG001-307 for type 1 SMA
Part of Brain & nervous system clinical trials.
This study follows up on patients with type 1 spinal muscular atrophy (SMA) who already received the EXG001-307 gene therapy in a previous trial. The goal is to monitor their long-term health and safety over time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have already received the EXG001-307 treatment in the earlier study (EXG001-307-102).
- A parent or guardian must understand the study and sign an informed consent form.
- You and your family must be willing to follow the study rules and attend all follow-up visits.
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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