Sly Syndrome
8
1
2
4
Key Insights
Highlights
Success Rate
80% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 37/100
12.5%
1 terminated out of 8 trials
80.0%
-6.5% vs benchmark
25%
2 trials in Phase 3/4
125%
5 of 4 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 4 completed trials
Clinical Trials (8)
Mucopolysaccharidosis VII Disease Monitoring Program
Expanded Access to Mepsevii
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
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
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism