MYOTONIC DYSTROPHY 1
Clinical trials for MYOTONIC DYSTROPHY 1 explained in plain language.
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Hope for DM1: Long-Term safety trial launches for experimental drug
Disease control Recruiting nowThis study looks at the long-term safety of an experimental drug called PGN-EDODM1 for people with myotonic dystrophy type 1 (DM1). It is for those who have already taken the drug in a previous study. About 48 adults will take part, and researchers will track any side effects ove…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE2 • Sponsor: PepGen Inc • Aim: Disease control
Last updated May 17, 2026 15:08 UTC
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New drug ATX-01 targets muscle stiffness in rare disease
Disease control Recruiting nowThis early-stage trial tests a new drug, ATX-01, in 56 adults aged 18 to 64 with myotonic dystrophy type 1 (DM1), a condition causing muscle stiffness and weakness. The main goal is to see if ATX-01 is safe and tolerable compared to a placebo. Participants will receive either one…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE1, PHASE2 • Sponsor: ARTHEx Biotech S.L. • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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New hope for muscle disease: experimental drug ARO-DM1 enters human trials
Disease control Recruiting nowThis study tests an experimental drug called ARO-DM1 in 78 adults with type 1 myotonic dystrophy, a genetic muscle disorder. The main goal is to see if the drug is safe and how it moves through the body. Participants receive either the drug or a placebo, and the study is still re…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE1, PHASE2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated May 11, 2026 20:40 UTC
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New drug trial aims to tame muscle stiffness in rare disease
Disease control Recruiting nowThis study tests an experimental drug called PGN-EDODM1 in 24 adults with myotonic dystrophy type 1 (DM1), a condition that causes muscle stiffness and weakness. The main goal is to see if the drug is safe and tolerable compared to a placebo. Researchers will also measure how the…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE2 • Sponsor: PepGen Inc • Aim: Disease control
Last updated May 06, 2026 16:03 UTC
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Common diabetes drug could ease muscle symptoms in rare disease
Symptom relief Recruiting nowThis study tests whether metformin, a common diabetes medicine, can improve muscle function in people with myotonic dystrophy type 1 (Steinert's disease). The trial will include 142 non-diabetic adults aged 18 to 70 who can still walk. Participants will receive either metformin o…
Matched conditions: MYOTONIC DYSTROPHY 1
Phase: PHASE3 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Symptom relief
Last updated May 04, 2026 16:31 UTC
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Massive spanish DM1 registry aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study is creating a large national registry in Spain for people with Myotonic Dystrophy Type 1 (DM1), a rare genetic muscle disease. Researchers will collect medical information, genetic data, and patient reports from up to 3,000 participants. The goal is to better understan…
Matched conditions: MYOTONIC DYSTROPHY 1
Sponsor: Fundació Institut Germans Trias i Pujol • Aim: Knowledge-focused
Last updated May 17, 2026 15:08 UTC