MYOTONIC DYSTROPHY
Clinical trials for MYOTONIC DYSTROPHY explained in plain language.
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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: MYOTONIC DYSTROPHY
Phase: PHASE1, PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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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: MYOTONIC DYSTROPHY
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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New study tracks Long-Term safety of muscle stiffness drug namuscla
Symptom relief CompletedThis study followed 53 adults with non-dystrophic myotonic disorders who were taking Namuscla (mexiletine) to manage muscle stiffness. Researchers monitored side effects and how often patients needed to reduce their dose or stop treatment over up to 36 months. The goal was to see…
Matched conditions: MYOTONIC DYSTROPHY
Sponsor: Lupin Ltd. • Aim: Symptom relief
Last updated Jun 27, 2026 14:01 UTC
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Heart risk study for muscular dystrophy patients completed
Knowledge-focused CompletedThis study looked at 537 people with myotonic dystrophy type 1 to understand their risk of serious heart rhythm problems, including sudden cardiac arrest. Researchers tracked participants for two years to see how often these events happened and what factors might predict them. Th…
Matched conditions: MYOTONIC DYSTROPHY
Phase: NA • Sponsor: Catholic University of the Sacred Heart • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:02 UTC