SPINAL MUSCULAR ATROPHY TYPE 2
Clinical trials for SPINAL MUSCULAR ATROPHY TYPE 2 explained in plain language.
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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…
Matched conditions: SPINAL MUSCULAR ATROPHY TYPE 2
Phase: PHASE3 • 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).…
Matched conditions: SPINAL MUSCULAR ATROPHY TYPE 2
Phase: PHASE3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:13 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…
Matched conditions: SPINAL MUSCULAR ATROPHY TYPE 2
Sponsor: Ohio State University • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:08 UTC