MYOTONIC DYSTROPHY
Clinical trials for MYOTONIC DYSTROPHY 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 DYSTROPHY
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 DYSTROPHY
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 07, 2026 18:42 UTC
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Heart risk study for muscle disease patients
Knowledge-focused CompletedThis study looked at 537 people with myotonic dystrophy type 1 to understand their risk of dangerous heart rhythms and sudden cardiac death. Researchers tracked participants for two years to see how many had major heart events. The goal was to find ways to predict who might be at…
Matched conditions: MYOTONIC DYSTROPHY
Phase: NA • Sponsor: Catholic University of the Sacred Heart • Aim: Knowledge-focused
Last updated May 15, 2026 11:53 UTC