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Clinical Trials for Mucopolysaccharidosis IV

• Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia
• A First-in-human Single and Repeated Dose Escalation Study of SAR442501 in Healthy Adults Subjects
• Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
• Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
• A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
• BMN 110 Phase 3B in Australian Patients
• BMN 110 US Expanded Access Program
• 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)
• Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
• A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
• A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)