MYOTONIC DYSTROPHY
Clinical trials for MYOTONIC DYSTROPHY explained in plain language.
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New hope for DM1: Long-Term trial of AOC 1001 underway
Disease control ENROLLING_BY_INVITATIONThis phase 3 study is testing the long-term safety and effectiveness of an experimental drug called AOC 1001 (del-desiran) for people with myotonic dystrophy type 1 (DM1), a genetic muscle disorder. The trial enrolls 230 adults who have completed a prior AOC 1001 study and will r…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Hope for muscle strength: new drug targets myotonic dystrophy
Disease control OngoingThis phase 3 trial tests an experimental drug called AOC 1001 in 159 adults with myotonic dystrophy type 1, a genetic condition that causes muscle weakness and stiffness. Participants receive either the drug or a placebo by intravenous infusion. The main goal is to see if the dru…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:12 UTC
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New test could predict falls in muscle disease patients
Diagnosis ENROLLING_BY_INVITATIONThis study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: LMU Klinikum • Aim: Diagnosis
Last updated Jun 26, 2026 16:30 UTC
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Hope for myotonic dystrophy: new drug shows promise in easing muscle stiffness Long-Term
Symptom relief ENROLLING_BY_INVITATIONThis study is testing the long-term safety and effectiveness of a drug called mexiletine PR for people with myotonic dystrophy types 1 and 2. The drug is taken once daily as a liquid to help reduce muscle stiffness (myotonia). The study includes 176 adults and teens who have alre…
Matched conditions: MYOTONIC DYSTROPHY
Phase: PHASE3 • Sponsor: Lupin Ltd. • Aim: Symptom relief
Last updated Jun 27, 2026 08:14 UTC