DM1
Clinical trials for DM1 explained in plain language.
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Experimental drug AOC 1001 tested for Long-Term safety in rare muscle disease
Disease control CompletedThis study is a follow-up to an earlier trial, testing the long-term safety and effects of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 participants who completed the first study received multiple doses of AOC 1001 by IV infu…
Matched conditions: DM1
Phase 2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Sep 10, 2026 00:00 UTC
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Hope for muscle strength: new drug targets myotonic dystrophy
Disease control CompletedThis 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: DM1
Phase 3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC
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New drug AOC 1001 tested for rare muscle disease
Disease control CompletedThis study tested a new drug called AOC 1001 in 39 adults with myotonic dystrophy type 1, a genetic muscle disorder. Participants received either the drug or a placebo by IV infusion. The goal was to check safety and how the drug moves through the body. Results will help decide i…
Matched conditions: DM1
Phase 1/2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC