MUSCULAR ATROPHY, SPINAL
Clinical trials for MUSCULAR ATROPHY, SPINAL explained in plain language.
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Daily medicine aims to stop devastating muscle disease in babies before it starts
Disease control OngoingThis study is testing a daily oral medicine called risdiplam in infants who have been diagnosed with spinal muscular atrophy (SMA) but do not yet show symptoms. The goal is to see if starting treatment very early can help control the disease and allow babies to reach important mo…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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Six-Year spinal injection study aims to confirm safety for rare muscle disease
Disease control OngoingThis study follows 115 people with spinal muscular atrophy (SMA) for nearly six years to learn about the long-term safety of a higher dose of the drug nusinersen. Participants, who previously took part in a related study, receive the drug via a spinal injection every four months.…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE3 • Sponsor: Biogen • Aim: Disease control
Last updated Mar 23, 2026 15:15 UTC
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China launches major study to track rare muscle disease in children
Knowledge-focused OngoingThis study is creating a national registry to understand how spinal muscular atrophy (SMA) naturally progresses in children under 18 in China. It will also track how and when children receive approved SMA treatments in real-world settings. The goal is to gather knowledge to help …
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Biogen • Aim: Knowledge-focused
Last updated Mar 31, 2026 12:12 UTC