MUSCULAR ATROPHY, SPINAL
Clinical trials for MUSCULAR ATROPHY, SPINAL explained in plain language.
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Can a spinal injection safely slow spinal muscular atrophy? a real-world study in korea seeks answers.
Disease control OngoingThis study tracks the safety and effectiveness of Spinraza (nusinersen) in people with spinal muscular atrophy (SMA) who receive the drug as part of routine care in Korea. Researchers will monitor for side effects and measure changes in motor skills over time. The goal is to see …
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Biogen • Aim: Disease control
Last updated Jul 29, 2026 00:00 UTC
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New drug BIIB115 aims to build on gene therapy for spinal muscular atrophy
Disease control OngoingThis early-stage trial tests a new drug called BIIB115 for spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The study first gives a single dose to healthy adult volunteers to check safety, then moves to children with SMA who have already received th…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE1 • Sponsor: Biogen • Aim: Disease control
Last updated Jul 11, 2026 00:00 UTC
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New drug shows promise in preventing SMA in newborns
Disease control OngoingThis study tests the drug risdiplam (Evrysdi) 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 can help them reach motor milestones like sitting without support. The trial involve…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Phase: PHASE2 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated Jul 11, 2026 00:00 UTC
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Can a nationwide registry unlock the secrets of adult spinal muscular atrophy?
Knowledge-focused OngoingThis study is a nationwide registry in China that will follow adults with a genetic form of spinal muscular atrophy (SMA). The goal is to describe how the disease naturally progresses over time and how disease-modifying treatments are used in real-world settings. By collecting da…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Biogen • Aim: Knowledge-focused
Last updated Aug 06, 2026 00:00 UTC
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New registry tracks SMA in chinese kids to unlock Real-World insights
Knowledge-focused OngoingThis study is a registry that collects information on up to 600 children in China with spinal muscular atrophy (SMA). It aims to describe how the disease progresses and how treatments are used in everyday medical practice. The study does not test a new drug but gathers data to be…
Matched conditions: MUSCULAR ATROPHY, SPINAL
Sponsor: Biogen • Aim: Knowledge-focused
Last updated Jun 26, 2026 12:32 UTC