Current and emerging management options for patients with Morquio A syndrome. Ther Clin Risk Manag 2013;9:45-53
Date
02/16/2013Pubmed ID
23413237Pubmed Central ID
PMC3572824DOI
10.2147/TCRM.S24771Scopus ID
2-s2.0-84875439601 (requires institutional sign-in at Scopus site) 25 CitationsAbstract
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.
