SMA
Clinical trials for SMA explained in plain language.
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New drug shows promise for kids with spinal muscular atrophy
Disease control CompletedThis study tested a new medicine called apitegromab in 188 children (ages 2–21) with later-onset spinal muscular atrophy (SMA types 2 and 3) who were already taking standard treatments. The goal was to see if adding apitegromab could further improve muscle function and movement. …
Matched conditions: SMA
Phase: PHASE3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated May 26, 2026 07:30 UTC
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One-Time gene therapy helps babies with rare muscle disease sit and breathe on their own
Disease control CompletedThis study tested a one-time gene therapy in 33 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment delivers a working copy of the missing SMN1 gene to help nerve cells survive. Results showed many babies could si…
Matched conditions: SMA
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated May 22, 2026 13:50 UTC