SPINAL MUSCULAR ATROPHY (SMA)
Clinical trials for SPINAL MUSCULAR ATROPHY (SMA) explained in plain language.
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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