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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One-Time gene therapy helps babies with deadly muscle disease sit and breathe on their own
Disease control CompletedThis phase 3 trial tested a one-time gene therapy called Zolgensma in 22 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment delivers a working copy of the missing SMN gene via an IV infusion. The main goals were …
Phase 3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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One-Time gene therapy helps babies with fatal muscle disease sit independently
Disease control CompletedThis study tested a one-time gene replacement therapy in 33 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment aims to replace the missing SMN1 gene to improve muscle function. Key results showed that many babies…
Phase 3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Jun 27, 2026 08:14 UTC
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New Muscle-Boosting drug shows promise for SMA patients in major trial
Disease control CompletedThis Phase 3 trial tested apitegromab, a drug that blocks a muscle-limiting protein, in 188 nonambulatory children and young adults with later-onset spinal muscular atrophy (SMA types 2 and 3). All participants were already taking standard SMA therapies (nusinersen or risdiplam).…
Phase 3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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French SMA questionnaire put to the test: will it measure up?
Knowledge-focused CompletedThis study looks at whether a French version of the SMAFRS questionnaire gives reliable results when used twice in adults with spinal muscular atrophy (SMA). About 60 participants will fill out the questionnaire during a routine visit and again 15 days later. No treatment is give…
Sponsor: Institut de Myologie, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC