Spinal muscular atrophy
Clinical trials for Spinal muscular atrophy explained in plain language.
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Can gene therapy help babies with SMA reach milestones? a Real-World review
Disease control Not yet recruitingThis study looks back at medical records of children with spinal muscular atrophy (SMA) type 1 who received a one-time gene therapy called onasemnogene abeparvovec (Zolgensma). Researchers want to see if treated infants can sit independently for 30 seconds or more within a year. …
Matched conditions: SPINAL MUSCULAR ATROPHY
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
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Can gene therapy zolgensma safely improve motor skills in SMA?
Knowledge-focused Not yet recruitingThis study follows 80 people with spinal muscular atrophy (SMA) in Japan who receive Zolgensma (onasemnogene abeparvovec) as an intrathecal injection. The goal is to track safety issues, such as liver problems or blood clotting abnormalities, and to measure changes in motor funct…
Matched conditions: SPINAL MUSCULAR ATROPHY
Sponsor: Novartis Pharmaceuticals • Aim: Knowledge-focused
Last updated Sep 19, 2026 00:00 UTC
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School transitions put to the test for children with a rare muscle disease
Knowledge-focused Not yet recruitingThis study uses questionnaires and focus groups to understand the challenges children with spinal muscular atrophy (SMA) and their families face when moving between schools or starting a new educational stage. Researchers aim to identify barriers to inclusion and support, and to …
Matched conditions: SPINAL MUSCULAR ATROPHY
Sponsor: Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust • Aim: Knowledge-focused
Last updated Jul 24, 2026 00:00 UTC