New gene therapy for SMA gets Real-World check in U.S. clinics
NCT ID NCT07448610
First seen Mar 26, 2026 · Last updated May 16, 2026 · Updated 4 times
Summary
This study follows 36 people with spinal muscular atrophy (SMA) who receive a one-time gene therapy (ITVISMA®) injected into the spine. Researchers will track how well it improves movement and what side effects occur in everyday medical practice. The goal is to see if this treatment works safely outside of controlled trials.
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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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