Mucopolysaccharidosis IVA
6
0
0
1
Key Insights
Highlights
No notable highlights available yet
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
66.7%
4 terminated out of 6 trials
20.0%
-66.5% vs benchmark
0%
0 trials in Phase 3/4
400%
4 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 (6)
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
BMN 110 US Expanded Access Program