Proximal spinal muscular atrophy
MONDO:0019079Proximal spinal muscular atrophies are a group of neuromuscular disorders characterized by progressive muscle weakness resulting from the degeneration and loss of the lower motor neurons in the spinal cord and the brain stem nuclei.
Also known as: SMA, Spinal Muscular Atrophy
57 clinical trials for this condition and its sub-types, 14 tagged with Proximal spinal muscular atrophy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Proximal spinal muscular atrophy
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Spinal muscular atrophy, type 1 17 trials
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Spinal muscular atrophy, type II 14 trials
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Spinal muscular atrophy, type III 13 trials
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Spinal muscular atrophy, type IV 2 trials
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3 sub-types
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Can a muscle-boosting antibody help people with spinal muscular atrophy over the long haul?
Disease control OngoingThis trial tests the long-term safety and effectiveness of apitegromab, an investigational antibody that blocks myostatin, a protein that limits muscle growth. People with type 2 or type 3 spinal muscular atrophy who completed earlier apitegromab studies can join. Researchers wil…
Phase 3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Sep 03, 2026 00:00 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…
Phase 3 • Sponsor: Biohaven Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Early access to experimental SMA drug apitegromab offered before approval
Disease control Expanded access (ended)This expanded access program provides apitegromab, an experimental drug that blocks myostatin to help maintain muscle strength, to eligible patients with spinal muscular atrophy (SMA) before it is officially approved. Participants must be at least 2 years old and have a confirmed…
Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC
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Can intensive therapy boost motor skills in kids with SMA?
Disease control OngoingThis pilot study tests whether combining intensive hand-arm and leg therapy with strength training can improve motor function in children with spinal muscular atrophy (SMA). Five children will attend one 6-hour session each weekend for 15 weeks. Researchers will check if the ther…
Sponsor: Teachers College, Columbia University • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC