Mucopolysaccharidosis IV A
8
1
1
5
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
0.0%
0 terminated out of 8 trials
100.0%
+13.5% vs benchmark
13%
1 trials in Phase 3/4
20%
1 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 5 completed trials
Clinical Trials (8)
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Natural History of Atypical Morquio A Disease
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Baby Detect : Genomic Newborn Screening
Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease