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Current and emerging management options for patients with Morquio A syndrome

Authors Algahim MF, Almassi GH

Received 14 November 2012

Accepted for publication 4 January 2013

Published 11 February 2013 Volume 2013:9 Pages 45—53

DOI https://doi.org/10.2147/TCRM.S24771

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Mohamed F Algahim, G Hossein Almassi

Division of Cardiothoracic Surgery, Medical College of Wisconsin, Milwaukee, WI, USA

Abstract: Morquio A syndrome is a lysosomal storage disease associated with mucopolysaccharidosis. It is caused by a deficiency of the lysosomal enzyme, N-acetylgalactosamine-6-sulfate sulfatase, which leads to accumulation of keratan sulfate and condroitin-6 sulfate in multiple organs. Patients present with multisystemic complications involving the musculoskeletal, respiratory, cardiovascular, and digestive systems. Presently, there is no definitive cure, and current management options are palliative. Enzyme replacement therapy and hematopoietic stem cell therapy have been proven effective in certain lysosomal storage diseases, and current investigations are underway to evaluate the effectiveness of these therapies and others for the treatment of Morquio A syndrome. This review discusses the current and emerging treatment options for Morquio A syndrome, citing examples of the treatment of other mucopolysaccharidoses.

Keywords: lysosomal, storage disease, mucopolysaccharidosis

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