Hurler-Scheie Syndrome
11
0
0
7
Key Insights
Highlights
Success Rate
70% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 42/100
27.3%
3 terminated out of 11 trials
70.0%
-16.5% vs benchmark
55%
6 trials in Phase 3/4
86%
6 of 7 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 7 completed trials
Clinical Trials (11)
RGX-111 Gene Therapy in Patients With MPS I
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I