MYOTONIC DYSTROPHY 1
Clinical trials for MYOTONIC DYSTROPHY 1 explained in plain language.
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First human trial tests potential new treatment for rare muscle disease
Disease control CompletedThis study tested the safety and effects of a new drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. Thirty-nine participants received either the drug or a placebo through an IV to see how their bodies handled it and if it affected th…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE1, PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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Drug tested to ease debilitating muscle disease in young people
Disease control CompletedThis study tested whether a drug called Tideglusib is safe and helpful for adolescents and adults with a severe, early-onset form of myotonic dystrophy, a genetic muscle-wasting disease. Sixteen participants aged 12-45 received the drug to see if it could improve muscle function,…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE2 • Sponsor: AMO Pharma Limited • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC
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Extended trial tests promising drug for genetic muscle disease
Disease control CompletedThis study continued testing an experimental drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. It followed 37 participants from a previous trial to check long-term safety and see if the drug continues to work when given every 8 weeks…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE2 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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First human test of potential muscle disease treatment
Disease control CompletedThis early-stage study tested the safety of a new drug called PGN-EDODM1 in 24 adults with Myotonic Dystrophy Type 1 (DM1), a genetic muscle-wasting disease. The main goal was to see if single doses given through an IV were safe and well-tolerated. Researchers also measured how t…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE1 • Sponsor: PepGen Inc • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC
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Hidden killer: study probes 10x higher blood clot risk in muscle disease
Knowledge-focused CompletedThis study aims to understand why people with myotonic dystrophy type 1 (DM1) have a much higher risk of dangerous blood clots in their veins. Researchers will test the blood of 130 participants (100 with DM1 and 30 healthy volunteers) to see if their blood clots too easily. They…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: NA • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Mar 31, 2026 12:11 UTC