SPINAL MUSCULAR ATROPHY
Clinical trials for SPINAL MUSCULAR ATROPHY explained in plain language.
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Gene therapy trial aims to halt Muscle-Wasting disease
Disease control OngoingThis study tests a gene therapy called vesemnogene lantuparvovec in about 20 people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The therapy delivers a working copy of the SMN1 gene to help muscles work better. The main goal is to check saf…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: PHASE1, PHASE2 • Sponsor: Lantu Biopharma • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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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 (SMA). About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The main goal is to…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: PHASE3 • Sponsor: Biohaven Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Higher dose of spinraza tested in SMA patients who already tried risdiplam
Disease control OngoingThis study tests a higher dose of nusinersen (Spinraza) in 45 teenagers and adults with spinal muscular atrophy (SMA) who cannot walk and have already taken risdiplam. Participants receive two 50 mg loading doses two weeks apart, then 28 mg every four months for about two years, …
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: PHASE3 • Sponsor: Biogen • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC
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SMA drug risdiplam under Long-Term watch: will it hold up?
Disease control OngoingThis study follows 403 adults and children with spinal muscular atrophy who are already taking risdiplam (Evrysdi) as prescribed by their doctor. Researchers will track side effects and how well the drug works over about two years. The goal is to see if risdiplam remains safe and…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: PHASE4 • Sponsor: Genentech, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:10 UTC
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SPINRAZA study tracks 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. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINR…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: NA • Sponsor: Centre Hospitalier Universitaire de Nice • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: SPINAL MUSCULAR ATROPHY
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 02:00 UTC
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New test could predict falls in muscle disease patients
Diagnosis ENROLLING_BY_INVITATIONThis study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …
Matched conditions: SPINAL MUSCULAR ATROPHY
Sponsor: LMU Klinikum • Aim: Diagnosis
Last updated Jun 26, 2026 16:30 UTC
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Can a 12-Week online course help babies with SMA develop better?
Symptom relief OngoingThis study tests a 12-week online program for mothers of infants (12-36 months old) with SMA Type 1. The program aims to teach mothers how to support their baby's development and improve their own knowledge. Thirteen mothers will take part, with 10 receiving the program and 3 rec…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: NA • Sponsor: Medipol University • Aim: Symptom relief
Last updated Jun 27, 2026 12:23 UTC
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Standing wheelchairs for kids: a tiny study hopes to open doors
Knowledge-focused OngoingThis study looks at whether a power wheelchair that can lift a child from sitting to standing is practical for kids with conditions like cerebral palsy, spinal cord injuries, or genetic diseases. Only 4 children aged 5-17 are taking part. The goal is to see if the chair helps the…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: NA • Sponsor: Grand Valley State University • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:22 UTC
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Wearable sensors track tiny movements in SMA babies on Cutting-Edge therapies
Knowledge-focused OngoingThis study follows 35 infants with spinal muscular atrophy (SMA) who are receiving gene therapy or other advanced treatments. Researchers use small wearable motion sensors to measure how well the babies move their arms and legs over two years. The goal is to create a more precise…
Matched conditions: SPINAL MUSCULAR ATROPHY
Phase: NA • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:15 UTC