MUSCULAR ATROPHY, SPINAL
Clinical trials for MUSCULAR ATROPHY, SPINAL explained in plain language.
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New drug shows promise in preventing SMA symptoms in babies
Disease control OngoingThis study tests an oral medicine called risdiplam in infants up to 6 weeks old who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The goal is to see if early treatment helps them reach motor milestones like sitting without support. 26 infants took…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated May 17, 2026 07:02 UTC
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New study tests higher dose of SMA drug over 6 years
Disease control OngoingThis study looks at the long-term safety of a higher dose of nusinersen for people with spinal muscular atrophy (SMA). It includes 115 participants who already completed a previous nusinersen study. They will receive the drug every 4 months for up to 64 months, and researchers wi…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE3 • Sponsor: Biogen • Aim: Disease control
Last updated May 13, 2026 16:01 UTC
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Chinese kids with SMA to be tracked in nationwide registry
Knowledge-focused OngoingThis study is a registry that collects information on up to 600 children in China who have spinal muscular atrophy (SMA). The goal is to learn how the disease progresses and how current treatments are used in real-world settings. No new treatment is given; the study simply observ…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Biogen • Aim: Knowledge-focused
Last updated May 13, 2026 15:59 UTC