Mucopolysaccharidosis Type I
7
1
2
4
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 7 trials
100.0%
+13.5% vs benchmark
0%
0 trials in Phase 3/4
25%
1 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 (7)
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
Biomarker for Hurler Disease (BioHurler)
MRS to Determine Neuroinflammation and Oxidative Stress in MPS I
Longitudinal Studies of Brain Structure and Function in MPS Disorders
Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III
Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI