MPS IVA
12
3
4
5
Key Insights
Highlights
Success Rate
71% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 47/100
16.7%
2 terminated out of 12 trials
71.4%
-15.1% vs benchmark
8%
1 trials in Phase 3/4
80%
4 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 (12)
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
Cardiac Structure and Function in MPS
Non-invasive Functional Assessment and Pathogenesis of Morquio A
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Gait Analysis in MPS IVA
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
Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
BMN 110 US Expanded Access Program