Spinal muscular atrophy, type III
MONDO:0009672Proximal spinal muscular atrophy type 3 (SMA3) is a relatively mild form of proximal spinal muscular atrophy characterized by muscle weakness and hypotonia resulting from the degeneration and loss of the lower motor neurons in the spinal cord and the brain stem nuclei.
Also known as: childhood spinal muscular atrophy, juvenile spinal muscular atrophy, Kugelberg Welander Syndrome, Kugelberg-Welander disease, SMA type 3, SMA type III, SMA-III, SMA3
28 clinical trials for this condition and its sub-types, 13 tagged with Spinal muscular atrophy, type III itself.
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New drug hopes to boost muscle power in babies with SMA
Disease control Recruiting nowThis study tests a drug called apitegromab in children under 2 years old who have spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The drug works by blocking a protein that limits muscle growth. Researchers want to see if adding apitegromab to stand…
Phase 2 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Sep 16, 2026 00:00 UTC
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Kids with chronic conditions walk stronger with VR at home
Symptom relief Recruiting nowThis study tests a new home-based program that uses a special treadmill and virtual reality to help children with conditions like cerebral palsy, muscle diseases, or obesity improve their walking. About 30 children aged 6 to 17 will try the program at home for at least 3 sessions…
Sponsor: University Hospital, Angers • Aim: Symptom relief
Last updated Jun 27, 2026 08:07 UTC
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New test aims to measure fatigue in SMA patients
Knowledge-focused Recruiting nowThis study is testing a new way to measure fatigue in people with spinal muscular atrophy (SMA), a condition that causes muscle weakness. The test is designed to be easy enough for patients with different levels of ability. Researchers want to see if the test is safe, doable, and…
Sponsor: Centre Hospitalier Universitaire de Saint Etienne • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:29 UTC
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Smart insoles could reveal hidden clues in rare childhood diseases
Knowledge-focused Recruiting nowThis study is testing whether special insoles worn inside shoes can measure how well children with Duchenne muscular dystrophy (DMD) or spinal muscular atrophy (SMA) walk. About 106 participants, including healthy children, will wear the insoles during walking tests and in daily …
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC
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Smart insoles could predict fracture risk in SMA patients
Knowledge-focused Recruiting nowThis study is looking at whether the way people with spinal muscular atrophy (SMA) walk can tell us about their bone health. Researchers will use special insoles to measure walking patterns and compare them with bone density scans and blood tests. The goal is to find simple, non-…
Sponsor: Jacqueline Montes • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:03 UTC