Gene therapy injection tested in Real-World fight against debilitating muscle disease
NCT ID NCT07448610
Summary
This study aims to gather real-world evidence on the safety and effectiveness of a one-time spinal injection gene therapy (ITVISMA®) for people with Spinal Muscular Atrophy (SMA). It will follow 36 U.S. participants, aged 2 and older, for at least a year after their treatment to see how their motor function and quality of life change. The study will also track side effects and how the treatment impacts healthcare needs like hospital visits and breathing support.
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