MYOTONIC DISORDERS
Clinical trials for MYOTONIC DISORDERS explained in plain language.
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New drug shows promise for rare muscle disease
Disease control CompletedThis study tested a new medicine called AOC 1001 in 39 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 the body processes it. Participants received either the drug or a place…
Matched conditions: MYOTONIC DISORDERS
Phase: PHASE1, PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 13, 2026 16:03 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