Hurler Syndrome
14
0
1
9
Key Insights
Highlights
Success Rate
90% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 25/100
7.1%
1 terminated out of 14 trials
90.0%
+3.5% vs benchmark
7%
1 trials in Phase 3/4
67%
6 of 9 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 9 completed trials
Clinical Trials (14)
RGX-111 Gene Therapy in Patients With MPS I
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
Neurobehavioral Phenotypes in MPS III
Stem Cell Transplant w/Laronidase for Hurler
Effects of Growth Hormone in Chronically Ill Children
Intrathecal Enzyme Replacement for Hurler Syndrome
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease