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Spinal muscular atrophy, type II
MONDO:0009673Proximal spinal muscular atrophy type 2 (SMA2) is a chronic infantile 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: Intermediate spinal muscular atrophy, SMA type 2, SMA type II, SMA-II, SMA2, chronic infantile spinal muscular atrophy, chronic spinal muscular atrophy, muscular atrophy, spinal, infantile chronic form
29 clinical trials for this condition and its sub-types, 14 tagged with Spinal muscular atrophy, type II 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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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