Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome

Background and Objectives Morquio A syndrome ( mucopolysaccharidosisIVA; MPS IVA) is a lysosomal storage disordercaused by deficiency of N-acetylgalactosamine-6-sulfatase, an enzyme required for degradation of the glycosaminoglycan keratan sulfate. Enzyme replacement therapywith elosulfase alfaprovides a potential therapy for Morquio A syndrome. We analyzed the pharmacokinetics and pharmacodynamicsof elosulfase alfain Morquio A patients from a phase III clinical trial.