MYOTONIC DISORDERS
Clinical trials for MYOTONIC DISORDERS 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 DISORDERS
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 DISORDERS
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:12 UTC