SMA
Clinical trials for SMA explained in plain language.
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One-Shot gene therapy offers hope for babies with devastating muscle disease
⭐️ CURE ⭐️ CompletedThis study tested a single-dose gene replacement therapy for infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe genetic disease that weakens muscles. The goal was to see if the one-time intravenous treatment could help babies achieve major milestones l…
Matched conditions: SMA
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: ⭐️ CURE ⭐️
Last updated Mar 18, 2026 14:40 UTC
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Muscle-Boosting drug shows promise for children with rare neuromuscular disease
Disease control CompletedThis completed Phase 3 trial tested whether adding the experimental drug apitegromab to standard SMA treatments could improve muscle function in 188 children and young adults (ages 2-21) with later-onset spinal muscular atrophy. All participants were already receiving approved SM…
Matched conditions: SMA
Phase: PHASE3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC