SMA
Clinical trials for SMA explained in plain language.
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New hope for babies with SMA: trial tests Muscle-Strengthening drug
Disease control Recruiting nowThis study is testing an investigational drug called apitegromab in infants under 2 years old who have spinal muscular atrophy (SMA). The main goal is to see how the drug moves through the body, how it affects a specific muscle protein, and if it can improve gross motor skills, l…
Matched conditions: SMA
Phase: PHASE2 • Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC
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Researchers test french SMA questionnaire for consistency
Knowledge-focused Recruiting nowThis study aims to check how reliable a French version of a questionnaire is for adults with spinal muscular atrophy (SMA). The questionnaire asks patients about their independence in daily activities like eating and walking. Participants will fill out the same questionnaire twic…
Matched conditions: SMA
Sponsor: Institut de Myologie, France • Aim: Knowledge-focused
Last updated Apr 04, 2026 03:06 UTC
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Swiss database tracks rare muscle diseases to speed up new treatments
Knowledge-focused Recruiting nowThis project is a national registry in Switzerland that collects health information from people with rare neuromuscular diseases like Duchenne muscular dystrophy and spinal muscular atrophy. It aims to connect patients to new treatment trials, help researchers understand these di…
Matched conditions: SMA
Sponsor: University of Bern • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:38 UTC