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 Aug 01, 2026 00:00 UTC
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New study to track gene Therapy's Real-World impact on spinal muscular atrophy
Disease control Not yet recruitingThis study will follow 36 people with spinal muscular atrophy (SMA) who receive a gene therapy called onasemnogene abeparvovec (ITVISMA) injected into the spinal fluid. The goal is to see how well it works and how safe it is in everyday medical practice. Participants must be at l…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: PHASE4 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 13:00 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