SMA
Clinical trials for SMA explained in plain language.
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One-Time gene therapy helps babies with fatal muscle disease sit independently
Disease control CompletedThis study tested a one-time gene replacement therapy in 33 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment aims to replace the missing SMN1 gene to improve muscle function. Key results showed that many babies…
Matched conditions: SMA
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: SMA
Phase: PHASE3 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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French SMA questionnaire put to the test: will it measure up?
Knowledge-focused CompletedThis study looks at whether a French version of the SMAFRS questionnaire gives reliable results when used twice in adults with spinal muscular atrophy (SMA). About 60 participants will fill out the questionnaire during a routine visit and again 15 days later. No treatment is give…
Matched conditions: SMA
Sponsor: Institut de Myologie, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC