Mucopolysaccharidosis VII
8
3
3
3
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 15/100
0.0%
0 terminated out of 8 trials
100.0%
+13.5% vs benchmark
13%
1 trials in Phase 3/4
67%
2 of 3 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (8)
Mucopolysaccharidosis VII Disease Monitoring Program
Registry of Patients Diagnosed With Lysosomal Storage Diseases
MPS (RaDiCo Cohort) (RaDiCo-MPS)
Expanded Access to Mepsevii
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Baby Detect : Genomic Newborn Screening
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age