MPS I
8
3
3
1
Key Insights
Highlights
Success Rate
33% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
25.0%
2 terminated out of 8 trials
33.3%
-53.2% vs benchmark
0%
0 trials in Phase 3/4
100%
1 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 1 completed trials
Clinical Trials (8)
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Longitudinal Study of Neurodegenerative Disorders
Cardiac Structure and Function in MPS
Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis