Glycogen Storage Disease Type II (GSD II)
6
0
0
5
Key Insights
Highlights
Success Rate
83% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 39/100
16.7%
1 terminated out of 6 trials
83.3%
-3.2% vs benchmark
50%
3 trials in Phase 3/4
60%
3 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 5 completed trials
Clinical Trials (6)
Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®
Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.