Mucopolysaccharidosis
8
0
0
5
Key Insights
Highlights
Success Rate
83% trial completion
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
12.5%
1 terminated out of 8 trials
83.3%
-3.2% vs benchmark
38%
3 trials in Phase 3/4
80%
4 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 (8)
The Effect of Enzyme Replacement Therapy in Mucopolysaccharidosis
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
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis