Pompe Disease Infantile-Onset
10
3
4
2
Key Insights
Highlights
Success Rate
67% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 49/100
10.0%
1 terminated out of 10 trials
66.7%
-19.8% vs benchmark
10%
1 trials in Phase 3/4
50%
1 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (10)
Registry of Patients Diagnosed With Lysosomal Storage Diseases
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
MSOT in Pompe Disease
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric 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
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies