MYOTONIC DYSTROPHY
Clinical trials for MYOTONIC DYSTROPHY explained in plain language.
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New drug aims to ease muscle stiffness in rare disease
Disease control OngoingThis phase 3 study tests an experimental drug called AOC 1001 for myotonic dystrophy type 1, a genetic condition that causes muscle weakness and stiffness. About 159 adults who can walk independently are receiving either the drug or a placebo to see if it improves hand function, …
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 26, 2026 06:45 UTC
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New hope for DM1: Long-Term trial of AOC 1001 begins
Disease control ENROLLING_BY_INVITATIONThis study is for people with myotonic dystrophy type 1 (DM1) who have already taken part in earlier AOC 1001 trials. It aims to see if the drug is safe and works well over a longer period. About 230 adults will receive the medicine by IV and be monitored for side effects and ben…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 22, 2026 13:52 UTC
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Hope for muscle stiffness: new drug shows promise in Long-Term trial
Symptom relief ENROLLING_BY_INVITATIONThis study looks at the long-term safety and effectiveness of a daily medication called mexiletine PR for people with myotonic dystrophy types 1 and 2, a condition that causes muscle stiffness. It is for 176 participants who have already completed a previous study on this drug. T…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Lupin Ltd. • Aim: Symptom relief
Last updated May 26, 2026 07:01 UTC
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New test aims to predict falls in muscle disease patients
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to create a simple test to determine the risk of falling for people with neuromuscular disorders like muscular dystrophy, ALS, and myasthenia gravis. Researchers will evaluate how well a series of physical tasks (like standing up, walking, and balancing) can predi…
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: LMU Klinikum • Aim: Knowledge-focused
Last updated May 26, 2026 06:57 UTC