Myotonic Disorders
7
1
3
3
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 7 trials
100.0%
+13.5% vs benchmark
29%
2 trials in Phase 3/4
0%
0 of 3 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (7)
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Global Study of Del-desiran for the Treatment of DM1
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders
Open-label Extension Study in Paediatric Patients Who Have Completed the MEX-NM-301 Study.
Characteristics of Nondystrophic Myotonias