MUSCULAR ATROPHY, SPINAL
Clinical trials for MUSCULAR ATROPHY, SPINAL explained in plain language.
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Early treatment may help babies with spinal muscular atrophy sit without support
Disease control OngoingThis study tested a daily oral medicine called risdiplam in 26 infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but do not yet show symptoms. The goal was to see if starting treatment early helps them reach motor milestones like sitting without support. Resul…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated May 26, 2026 05:23 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 (a drug for spinal muscular atrophy) in about 115 people who already completed a previous nusinersen study. Participants will receive the drug every 4 months via a lumbar puncture for up to 64 months, with fo…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE3 • Sponsor: Biogen • Aim: Disease control
Last updated May 26, 2026 04:35 UTC
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600 kids with spinal muscular atrophy to be tracked in landmark chinese study
Knowledge-focused OngoingThis study is a nationwide registry in China that follows 600 children with spinal muscular atrophy (SMA). The goal is to understand how the disease progresses naturally and how well current treatments work in everyday life. Researchers will collect information from both past med…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Biogen • Aim: Knowledge-focused
Last updated May 26, 2026 05:30 UTC