Spinal muscular atrophy patient registry for OAV-101
Part of Brain & nervous system clinical trials.
This is a registry that collects information from people with spinal muscular atrophy (SMA) who have been treated with OAV-101. It helps researchers understand how patients do over time after this treatment.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a genetically confirmed diagnosis of spinal muscular atrophy (SMA).
- You have been treated with OAV-101 (Zolgensma).
- You (or your legal guardian) can provide consent, and if needed, you can agree as well (assent).
- You are not currently taking part in another SMA treatment study that uses an investigational drug.
- If you are on an expanded access/compassionate use program for OAV-101 (like MAP/EAP/SPI/NPP), you can still join.
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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