Mucopolysaccharidosis IVA
Pipeline by Development Stage
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (4)
Total enrollment: 56 patients across 4 trials
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
BMN 110 US Expanded Access Program
Phase Legend
Key Insights
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