Lysosomal Storage Disease
10
0
0
8
Key Insights
Highlights
Success Rate
89% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
10.0%
1 terminated out of 10 trials
88.9%
+2.4% vs benchmark
0%
0 trials in Phase 3/4
25%
2 of 8 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 8 completed trials
Clinical Trials (10)
Study of Inborn Errors of Cholesterol Synthesis and Related Disorders
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
Gene Therapy for Metachromatic Leukodystrophy (MLD)
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
The Classification and Cause of Leukodystrophies of Unknown Cause
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
Magnetic Stimulation of the Human Nervous System