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A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A...

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)

This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with a...

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 (Prism301)

The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of...

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

AGT-182 is a fusion protein containing idursulfase that is intended to deliver the enzyme peripheral...

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