SMA
Clinical trials for SMA explained in plain language.
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New drug shows promise for muscle strength in SMA patients on standard therapy
Disease control CompletedThis study tested a drug called apitegromab in 188 people aged 2 to 21 with later-onset spinal muscular atrophy (SMA types 2 and 3) who were already taking nusinersen or risdiplam. The goal was to see if adding apitegromab could improve muscle function more than a placebo. Partic…
Matched conditions: SMA
Phase: PHASE3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated May 14, 2026 12:05 UTC
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Single infusion offers hope for babies with devastating muscle disease
Disease control CompletedThis study tested a one-time gene therapy for babies under 6 months old with spinal muscular atrophy type 1, a severe genetic disease that weakens muscles. The therapy aimed to deliver a working copy of a missing gene through a single intravenous infusion. Researchers measured wh…
Matched conditions: SMA
Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: Disease control
Last updated Apr 07, 2026 14:35 UTC