SPINAL MUSCULAR ATROPHY TYPE I
Clinical trials for SPINAL MUSCULAR ATROPHY TYPE I explained in plain language.
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Gene therapy hope for babies with rare muscle disease
Disease control Recruiting nowThis study tests a single dose of GB221, a gene therapy that delivers a working SMN1 gene, in infants aged 2 weeks to under 12 months with spinal muscular atrophy (SMA) Type 1. The goal is to see if it is safe and helps improve motor function. The trial includes both symptomatic …
Matched conditions: SPINAL MUSCULAR ATROPHY TYPE I
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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New study tracks Risdiplam's Real-World impact on spinal muscle atrophy
Knowledge-focused Recruiting nowThis study will observe 30 children with type I or type II spinal muscle atrophy who are taking risdiplam. Researchers will track motor function improvements over 24 months using standard tests. The goal is to see how well the drug works in everyday medical practice.
Matched conditions: SPINAL MUSCULAR ATROPHY TYPE I
Sponsor: Hoffmann-La Roche • Aim: Knowledge-focused
Last updated Aug 06, 2026 00:00 UTC