SPINAL MUSCULAR ATROPHY
Clinical trials for SPINAL MUSCULAR ATROPHY explained in plain language.
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New drug shows promise for SMA patients in major trial
Disease control OngoingThis study tests whether adding taldefgrobep alfa to standard SMA treatments can improve muscle function in people with spinal muscular atrophy. About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The goal is to see if the…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: PHASE3 • Sponsor: Biohaven Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 17, 2026 15:16 UTC
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New study monitors SPINRAZA's effect on movement in adults with spinal muscular atrophy
Disease control OngoingThis study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA, a medication given via spinal injection. Researchers will measure changes in motor function, like sitting, standing, and arm movement, over 27 months. The goal is to see how w…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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New study tracks SMA patients on risdiplam for two years
Disease control OngoingThis study follows about 400 adults and children with spinal muscular atrophy (SMA) who are already taking or starting risdiplam (Evrysdi). Researchers will monitor them for about 2 years to see how safe the drug is and how well it works over time. The goal is to understand long-…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: PHASE4 • Sponsor: Genentech, Inc. • Aim: Disease control
Last updated May 13, 2026 15:58 UTC
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New drug aims to boost walking ability in adults with muscle-weakening disease
Symptom relief OngoingThis study tests an experimental drug called NMD670 in 54 adults with type 3 spinal muscular atrophy, a condition that causes muscle weakness. Participants take the drug or a placebo for a period, then switch, to see if the drug improves walking distance and muscle strength. The …
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: PHASE2 • Sponsor: NMD Pharma A/S • Aim: Symptom relief
Last updated May 15, 2026 11:54 UTC
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Wearable sensors track tiny triumphs in SMA babies
Knowledge-focused OngoingThis study aims to better measure how well infants with spinal muscular atrophy (SMA) can move after receiving new treatments like gene therapy. Researchers will use small wearable sensors on the babies' arms and legs to track changes in movement over time. The goal is to create …
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: NA • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 17, 2026 15:15 UTC
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New test could predict falls in muscle disease patients
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to create a simple test to predict fall risk in people with neuromuscular disorders like muscular dystrophy, ALS, and myasthenia gravis. Researchers will measure how well participants perform tasks like standing up from a chair, walking, and balancing. The goal is…
Matched conditions: SPINAL MUSCULAR ATROPHY
Sponsor: LMU Klinikum • Aim: Knowledge-focused
Last updated May 07, 2026 18:39 UTC
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Standing wheelchairs for kids: a small study looks at big possibilities
Knowledge-focused OngoingThis study looks at whether a powered wheelchair that can lift a child to a standing position helps them take part in more activities. It includes children aged 5 to 17 with conditions like cerebral palsy, spinal cord injuries, or genetic disorders who cannot walk. The goal is to…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: NA • Sponsor: Grand Valley State University • Aim: Knowledge-focused
Last updated May 07, 2026 18:39 UTC
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New study: online coaching for moms of SMA toddlers shows promise
Knowledge-focused OngoingThis study looks at whether a 12-week online program can help mothers of toddlers (12-36 months) with spinal muscular atrophy type 1 learn more about child development and support their child's growth. The program involves 13 mothers, with 10 receiving the weekly 90-minute sessio…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: NA • Sponsor: Medipol University • Aim: Knowledge-focused
Last updated Apr 30, 2026 15:52 UTC