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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Gene therapy breakthrough: one dose may help babies with rare muscle disease
Disease control CompletedThis study tested a single dose of Zolgensma gene therapy in 30 infants diagnosed with spinal muscular atrophy (SMA) before symptoms appeared. The goal was to see if the treatment helps them reach motor milestones like sitting or standing alone. The therapy delivers a working cop…
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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Gene therapy shows promise for kids with SMA type 2
Disease control CompletedThis study tested a one-time gene therapy called OAV101 in 126 children aged 2 to 18 with type 2 spinal muscular atrophy (SMA) who had never received other SMA treatments. The therapy was given as a spinal injection and compared to a sham procedure. The goal was to see if it coul…
Phase 3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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New fabric brace could give arm strength back to patients with muscle diseases
Symptom relief CompletedThis study tests a special fabric shoulder brace designed to mimic muscle support for people with neuromuscular disorders like muscular dystrophy, SMA, and ALS. About 30 participants will wear the brace and perform arm tasks to see if it improves movement, strength, and daily fun…
Sponsor: Seoul National University Hospital • Aim: Symptom relief
Last updated Sep 05, 2026 00:00 UTC
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New knee device may help kids with SMA build leg strength
Symptom relief CompletedThis study tested a portable knee training device in 13 children aged 6-12 with spinal muscular atrophy (SMA). The goal was to see if regular exercise with the device could improve leg muscle strength and function. Researchers measured muscle size, strength, and nerve activity ov…
Sponsor: Peking University Third Hospital • Aim: Symptom relief
Last updated Jun 27, 2026 12:36 UTC
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New tests could reveal how SMA progresses in adults
Knowledge-focused CompletedThis study looked at 67 adults with spinal muscular atrophy (SMA) types 2 and 3 to see if special electrical tests (MUNE and CMAP) can track nerve loss over time. The goal was to find better ways to measure how the disease changes, not to test a treatment. Participants had geneti…
Sponsor: Ohio State University • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:08 UTC
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Braces and SMA: new insights on sitting, standing, and walking
Knowledge-focused CompletedThis study looked back at 80 children with spinal muscular atrophy (SMA) types II and III to see how using braces (orthoses) helped them sit, stand, and walk. Researchers recorded when and how often children achieved these milestones and what types of braces they used. The goal w…
Sponsor: Azienda USL Reggio Emilia - IRCCS • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:06 UTC