Myotonic Dystrophy Type 1 (DM1)
17
6
9
6
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
0.0%
0 terminated out of 17 trials
100.0%
+13.4% vs benchmark
12%
2 trials in Phase 3/4
17%
1 of 6 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 6 completed trials
Clinical Trials (17)
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
Personalized Training for People With Rare Neuromuscular Disorders
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Remote Assessments and Genetic Determinants of Myotonic Dystrophy
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Global Study of Del-desiran for the Treatment of DM1
A Registered Observational Cohort Study of Myotonic Dystrophy Type 1
A Remote Physical Activity Program in the Population Suffering from Type 1 Myotonic Dystrophy
Technology Assisted Rehabilitation for Upper Limb Function in Myotonic Dystrophy Type 1
Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders
Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Brain Involvement in Myotonic Dystrophy Type I: From Functional Neuroimaging to the Impact on Quality of Life