MYOTONIC DYSTROPHY 1
Clinical trials for MYOTONIC DYSTROPHY 1 explained in plain language.
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Experimental drug aims to control debilitating muscle disease
Disease control OngoingThis global Phase 3 trial is testing whether an intravenous drug called AOC 1001 can help control symptoms of myotonic dystrophy type 1, a genetic muscle-wasting disease. About 159 adults with a confirmed diagnosis will receive either the drug or a placebo via infusion every 8 we…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC
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Four-Year trial tests potential new treatment for debilitating muscle disease
Disease control ENROLLING_BY_INVITATIONThis study is testing the long-term safety and effectiveness of an investigational drug called del-desiran for adults with myotonic dystrophy type 1 (DM1), a genetic muscle-wasting disease. Participants who completed a previous related study will receive an intravenous infusion o…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC
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Scientists map the journey of a rare muscle disease
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to better understand Myotonic Dystrophy Type 1 (DM1) in Chinese patients by observing how the disease progresses over time. Researchers will follow 500 adult patients, collecting detailed health information through tests, scans, and questionnaires. The goal is to …
Matched conditions: MYOTONIC DYSTROPHY 1
Sponsor: Huashan Hospital • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC