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New study tracks gene Therapy's Real-World impact on SMA patients

NCT ID NCT07448610

First seen Mar 26, 2026 · Last updated May 22, 2026 · Updated 6 times

Summary

This study looks at how safe and effective a gene therapy called onasemnogene abeparvovec (ITVISMA®) is for people with spinal muscular atrophy (SMA) in everyday medical practice. It will include 36 participants aged 2 and older who receive the treatment as part of their routine care. Researchers will track changes in motor function and any side effects over time.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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