MYOTONIC DYSTROPHY
Clinical trials for MYOTONIC DYSTROPHY explained in plain language.
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First human trial tests potential new treatment for rare muscle disease
Disease control CompletedThis study tested the safety and effects of a new drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. Thirty-nine participants received either the drug or a placebo through an IV to see how their bodies handled it and if it affected th…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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Extended trial tests promising drug for genetic muscle disease
Disease control CompletedThis study continued testing an experimental drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. It followed 37 participants from a previous trial to check long-term safety and see if the drug continues to work when given every 8 weeks…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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Researchers track deadly heart risks in muscle disease patients
Knowledge-focused CompletedThis study aimed to understand why people with myotonic dystrophy type 1 (a muscle-wasting disease) are at high risk for sudden cardiac death. Researchers followed 537 adult patients across Italy for two years, using heart tests and monitoring devices to identify the specific hea…
Matched conditions: MYOTONIC DYSTROPHY
Phase: NA • Sponsor: Catholic University of the Sacred Heart • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:31 UTC