Study Summary
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S. Pragmatic Multicenter Study (STREAM).
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Onasemnogene Abeparvovec-brveDRUG
administered once via lumbar puncture with systemic corticosteroid prophylaxis per label.
Study Locations
No locations listed.