Mucopolysaccharidosis Type II
7
1
1
6
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
17%
1 of 6 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 6 completed trials
Clinical Trials (7)
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers
Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II
A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers
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