MYOTONIC DYSTROPHY 1
Clinical trials for MYOTONIC DYSTROPHY 1 explained in plain language.
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Experimental drug tideglusib tested for rare muscle disease
Disease control CompletedThis study tested a drug called tideglusib in 16 adolescents and adults with a rare muscle disease called myotonic dystrophy type 1. The goal was to see if the drug is safe and if it helps with symptoms. Participants took either 400 mg or 1000 mg of tideglusib daily, and research…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE2 • Sponsor: AMO Pharma Limited • Aim: Disease control
Last updated May 17, 2026 14:58 UTC
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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 1
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 1
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated May 07, 2026 18:42 UTC
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New drug shows promise for muscle stiffness in rare disease
Symptom relief CompletedThis early-stage study tested a single dose of a new drug called PGN-EDODM1 in 24 adults with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The main goal was to check if the drug is safe and how the body processes it. Researchers monitore…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE1 • Sponsor: PepGen Inc • Aim: Symptom relief
Last updated May 11, 2026 20:41 UTC
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Hidden danger: muscle disease linked to deadly clots
Knowledge-focused CompletedThis study investigates why people with myotonic dystrophy type 1 (DM1) face a much higher risk of dangerous blood clots in the legs or lungs. Researchers will test blood clotting and gene activity in 130 participants, including those with DM1, clot history, and healthy volunteer…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: NA • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 12, 2026 13:41 UTC