Myotonic dystrophy 1
Clinical trials for Myotonic dystrophy 1 explained in plain language.
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New hope for DM1: Long-Term trial of AOC 1001 underway
Disease control By invitation onlyThis 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 1
Phase 3 • Sponsor: Avidity Biosciences, Inc. • Aim: Disease control
Last updated Aug 21, 2026 00:00 UTC
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New study tracks how DM1 affects chinese patients over time
Knowledge-focused By invitation onlyThis study follows 1000 Chinese patients with myotonic dystrophy type 1 (DM1) to understand how the disease affects multiple body systems and leads to disability. Researchers will collect health data and blood samples every 3 to 6 months for several years. The goal is to identify…
Matched conditions: MYOTONIC DYSTROPHY 1
Sponsor: Huashan Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:00 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 1
Sponsor: University Hospital, Lille • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:36 UTC