Hendriksz, Christian J.2016-12-062016-11Hendriksz, CJ 2016, 'Elosulfase alfa (BMN 110) for the treatment of Mucopolysaccharidosis IVA (Morquio A syndrome)', Expert Review of Clinical Pharmacology, vol. 9, no. 12, pp. 1521-1532.1751-2433 (print)1751-2441 (online)10.1080/17512433.2017.1260000http://hdl.handle.net/2263/58352INTRODUCTION : Morquio A syndrome is a rare, autosomal recessive, lysosomal storage disorder caused bya deficiency in the enzyme N-acetylgalactosamine-6-sulfatase (GALNS). In 2014, the use of recombinant human GALNS, elosulfase alfa, was approved in Europe, Canada, the United States, Australia, and Brazil for the treatment of Morquio A syndrome. Elosulfase alfa is administered intravenously once-weekly at a dose of 2.0 mg/kg. AREAS COVERED : This is a review of the efficacy, safety and tolerability, pharmacokinetics and pharmacodynamics, and other outcomes of elosulfase alfa treatment of patients with Morquio A. A discussion of other treatment considerations, limitations, and future directions in the use of elosulfase alfa is provided. EXPERT COMMENTARY : Pharmacokinetic studies outside of clinical trials and in “real-world” clinical settings need to be performed. We cannot currently predict which patient is going to respond well to enzyme replacement therapy; thus, all patients should be given the option to receive treatment for at least 12 months. Additionally, accurate biomarkers for evaluating disease state and drug responsiveness would greatly aid in the treatment of patients with Morquio A. In addition, improved and innovative daily lifestyle measures are greatly needed to adequately measure clinical response and true impact on quality of life.en© Taylor and Francis. This is an electronic version of an article published in Expert Review of Clinical Pharmacology, vol. 9, no. 12, pp. 1521-1532, 2016. doi : 10.1080/17512433.2017.1260000. Expert Review of Clinical Pharmacology is available online at : http://tandfonline.com/toc/ierj20.Enzyme replacement therapyGALNSLysosomal storage disorderMPS IVAMucopolysaccharidosisMorquio A syndromeElosulfase alfa treatmentBya deficiencyEfficacySafety and tolerabilityPharmacokineticsPharmacodynamicsPostprint Article