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)
Non-invasive Functional Assessment and Pathogenesis of Morquio A
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
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