Congenital Myasthenic Syndrome
11
4
4
4
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 11 trials
100.0%
+13.5% vs benchmark
0%
0 trials in Phase 3/4
0%
0 of 4 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 4 completed trials
Clinical Trials (11)
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution
Baby Detect : Genomic Newborn Screening
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia
Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes
3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes
Pregnancy Outcomes in Congenital Myasthenie Syndrome