MYOTONIC DISORDERS
Clinical trials for MYOTONIC DISORDERS explained in plain language.
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New drug shows promise in early trial for rare muscle disease
Disease control CompletedThis study tested a new medicine called AOC 1001 in 38 adults with myotonic dystrophy type 1 (DM1), a genetic disease that causes muscle weakness and stiffness. The goal was to see if the drug is safe and how it works in the body. Participants received either the drug or a placeb…
Matched conditions: MYOTONIC DISORDERS
Phase: PHASE1, PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 22, 2026 14:03 UTC
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Experimental DM1 drug shows promise in long-term safety trial
Disease control CompletedThis study looked at the long-term safety of a drug called AOC 1001 in adults with myotonic dystrophy type 1 (DM1), a genetic muscle disease. 37 people who completed an earlier study received multiple doses of the drug by IV. Researchers tracked side effects and measured drug lev…
Matched conditions: MYOTONIC DISORDERS
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 07, 2026 18:42 UTC