MYOTONIC DYSTROPHY
Clinical trials for MYOTONIC DYSTROPHY explained in plain language.
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Hope for muscle disease: new drug enters final testing phase
Disease control OngoingThis study tests a new medicine called AOC 1001 for people with myotonic dystrophy type 1, a genetic disease that causes muscle weakness and stiffness. About 159 adults with a confirmed diagnosis will receive either the drug or a placebo by IV. The main goal is to see if the drug…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 14, 2026 12:02 UTC
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Hope for DM1: new drug tested for long-term safety
Disease control ENROLLING_BY_INVITATIONThis study looks at the long-term safety and effects of a drug called AOC 1001 in people with myotonic dystrophy type 1 (DM1), a genetic muscle disease. About 230 adults who already took part in earlier AOC 1001 studies will receive the drug intravenously. The main goal is to tra…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 11, 2026 20:49 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 reducing muscle stiffness (myotonia) in people with myotonic dystrophy types 1 and 2. It is for those who have already completed a previous study of the same drug. The goal i…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Lupin Ltd. • Aim: Symptom relief
Last updated May 15, 2026 11:56 UTC
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New test could predict falls in muscle disease patients
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to create a simple test to predict fall risk in people with neuromuscular disorders like muscular dystrophy, ALS, and myasthenia gravis. Researchers will measure how well participants perform tasks like standing up from a chair, walking, and balancing. The goal is…
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: LMU Klinikum • Aim: Knowledge-focused
Last updated May 07, 2026 18:39 UTC