DM1
10
5
5
4
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 30/100
0.0%
0 terminated out of 10 trials
100.0%
+13.4% vs benchmark
30%
3 trials in Phase 3/4
25%
1 of 4 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 4 completed trials
Clinical Trials (10)
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
Global Study of Del-desiran for the Treatment of DM1
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Remote Assessments and Genetic Determinants of Myotonic Dystrophy
The Spanish National Registry for Myotonic Dystrophy Type 1
Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients