Real-world study of Zolgensma for SMA type 1 in Saudi Arabia
Part of Brain & nervous system clinical trials.
This study reviews medical records of people with spinal muscular atrophy type 1 who received the gene therapy Zolgensma (onasemnogene abeparvovec) to understand their treatment journey and outcomes. It may help doctors improve care for future patients.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of SMA type 1.
- You must have been treated with the gene therapy Zolgensma.
- You need at least one doctor visit before starting treatment and two visits after.
- Your treatment must have started in January 2023 or later.
- You must have been followed for at least 3 months after receiving the therapy.
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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