MYOTONIC DYSTROPHY TYPE 1
Clinical trials for MYOTONIC DYSTROPHY TYPE 1 explained in plain language.
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New hope for DM1: Long-Term trial of AOC 1001 underway
Disease control ENROLLING_BY_INVITATIONThis phase 3 study is testing the long-term safety and effectiveness of an experimental drug called AOC 1001 (del-desiran) for people with myotonic dystrophy type 1 (DM1), a genetic muscle disorder. The trial enrolls 230 adults who have completed a prior AOC 1001 study and will r…
Matched conditions: MYOTONIC DYSTROPHY TYPE 1
Phase: PHASE3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Hope for myotonic dystrophy: new drug shows promise in easing muscle stiffness Long-Term
Symptom relief ENROLLING_BY_INVITATIONThis study is testing the long-term safety and effectiveness of a drug called mexiletine PR for people with myotonic dystrophy types 1 and 2. The drug is taken once daily as a liquid to help reduce muscle stiffness (myotonia). The study includes 176 adults and teens who have alre…
Matched conditions: MYOTONIC DYSTROPHY TYPE 1
Phase: PHASE3 • Sponsor: Lupin Ltd. • Aim: Symptom relief
Last updated Jun 27, 2026 08:14 UTC
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Brain scans reveal diabetes link to cognitive decline in rare disease
Knowledge-focused OngoingThis study looks at whether diabetes or blood sugar problems can worsen thinking and memory issues in people with myotonic dystrophy type 1. Researchers will use MRI brain scans and thinking tests over 4 years to track changes. The goal is to understand why some patients have mor…
Matched conditions: MYOTONIC DYSTROPHY TYPE 1
Sponsor: University Hospital, Lille • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:36 UTC
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New study aims to uncover why people with muscle disease fall
Knowledge-focused OngoingThis study measures how often people with myotonic dystrophy type 1 fall and what factors affect their balance. Researchers will also assess fear of falling and leg muscle strength. The goal is to better understand fall risk in this condition.
Matched conditions: MYOTONIC DYSTROPHY TYPE 1
Sponsor: Antalya Training and Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC