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- Title
Current and emerging management options for patients with Morquio A syndrome.
- Authors
Algahim, Mohamed F; Almassi, G Hossein
- 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.
- Publication
Therapeutics and clinical risk management, 2013, Vol 9, p45
- ISSN
1176-6336
- Publication type
Journal Article
- DOI
10.2147/TCRM.S24771