SPINAL MUSCULAR ATROPHY (SMA)
Clinical trials for SPINAL MUSCULAR ATROPHY (SMA) explained in plain language.
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Zolgensma's long-term effects under the microscope in new 5-year study
Disease control OngoingThis study follows 175 people with spinal muscular atrophy who previously received the gene therapy Zolgensma in clinical trials. Researchers will monitor them for 5 years to track serious side effects and developmental milestones. The goal is to understand how safe and effective…
Matched conditions: SPINAL MUSCULAR ATROPHY (SMA)
Phase: PHASE3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jul 11, 2026 00:00 UTC
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New Muscle-Boosting drug combo tested for SMA
Disease control OngoingThis study tests whether adding an experimental drug (RO7204239) to an existing SMA medicine (risdiplam) can help people with spinal muscular atrophy build stronger muscles and move better. The trial includes about 259 children and young adults, ages 2 to 25, who can walk. Resear…
Matched conditions: SPINAL MUSCULAR ATROPHY (SMA)
Phase: PHASE2, PHASE3 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Jun 27, 2026 13:07 UTC
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Gene therapy hope for SMA kids: early trial launches
Disease control OngoingThis early-stage trial tests a new gene therapy called NKG001 in 21 children under 5 with spinal muscular atrophy (SMA). The therapy is given as a single dose, either through a vein or combined with a spinal injection. The main goal is to check safety and find the best dose, not …
Matched conditions: SPINAL MUSCULAR ATROPHY (SMA)
Phase: NA • Sponsor: Nikegen Pharmaceutical (Hangzhou) Company Limited • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC