Late-Onset Pompe Disease
6
1
1
2
Key Insights
Highlights
Success Rate
40% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 65/100
50.0%
3 terminated out of 6 trials
40.0%
-46.6% vs benchmark
17%
1 trials in Phase 3/4
100%
2 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 2 completed trials
Clinical Trials (6)
LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONS
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
High Dose Inspiratory Muscle Training in LOPD
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)