MPS VI
5
1
2
2
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 5 trials
100.0%
+13.5% vs benchmark
0%
0 trials in Phase 3/4
0%
0 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 (5)
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02