MYOTONIC DISORDERS
Clinical trials for MYOTONIC DISORDERS 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 DISORDERS
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 26, 2026 05:44 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 DISORDERS
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 22, 2026 13:52 UTC