Spinal muscular atrophy, type 1
MONDO:0009669A severe infantile form of proximal spinal muscular atrophy characterized by severe and progressive 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: infantile muscular atrophy, infantile spinal muscular atrophy, spinal muscular atrophies of childhood, SMA type 1, SMA type I, SMA-I, SMA1, SMNI
32 clinical trials for this condition and its sub-types, 17 tagged with Spinal muscular atrophy, type 1 itself.
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Gene therapy hope for babies with fatal muscle disease
Disease control Recruiting nowThis early-phase trial tests a gene therapy called SKG0201 in 11 infants with spinal muscular atrophy (SMA) type I, a severe muscle-weakening disease. The therapy uses a harmless virus to deliver a working copy of the missing SMN1 gene. The study aims to see if it is safe and can…
Phase 1/2 • Sponsor: Lanyue Biotech (Hangzhou) Co., Ltd. • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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Gene therapy hope for babies with rare muscle disease
Disease control Recruiting nowThis study tests a single dose of GB221, a gene therapy that delivers a working SMN1 gene, in infants aged 2 weeks to under 12 months with spinal muscular atrophy (SMA) Type 1. The goal is to see if it is safe and helps improve motor function. The trial includes both symptomatic …
Phase 1/2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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New study tracks Risdiplam's Real-World impact on spinal muscle atrophy
Knowledge-focused Recruiting nowThis study will observe 30 children with type I or type II spinal muscle atrophy who are taking risdiplam. Researchers will track motor function improvements over 24 months using standard tests. The goal is to see how well the drug works in everyday medical practice.
Sponsor: Hoffmann-La Roche • Aim: Knowledge-focused
Last updated Sep 05, 2026 00:00 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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Bath time breakthrough: new test spots hidden movements in SMA babies
Knowledge-focused Recruiting nowThis study aims to improve how doctors assess movement in infants with spinal muscular atrophy (SMA), a severe muscle-weakening disease. Researchers will use sensors to measure babies' movements in a special bathtub, where water helps reduce gravity's effects. The goal is to crea…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:59 UTC