Pompe's Disease
6
1
1
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 6 trials
100.0%
+13.5% vs benchmark
33%
2 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 (6)
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth
Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana
Prevalence of Pompe's Disease in Respiratory Clinics
Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis