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

BACKGROUND AND OBJECTIVES: Morquio A syndrome (mucopolysaccharidosis IVA; MPS IVA) is a lysosomal storage disorder caused by deficiency of N-acetylgalactosamine-6-sulfatase, an enzyme required for degradation of the glycosaminoglycan keratan sulfate. Enzyme replacement therapy with elosulfase alfa p...

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Publicat a:Clin Pharmacokinet
Autors principals: Qi, Yulan, Musson, Donald G., Schweighardt, Becky, Tompkins, Troy, Jesaitis, Lynne, Shaywitz, Adam J., Yang, Ke, O’Neill, Charles A.
Format: Artigo
Idioma:Inglês
Publicat: Springer International Publishing 2014
Matèries:
Original Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4243006/
https://ncbi.nlm.nih.gov/pubmed/25234648
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40262-014-0173-y
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