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KMID : 1146120170030020037
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Journal of Mucopolysaccharidosis and Rare Diseases
2017 Volume.3 No. 2 p.37 ~ p.40
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A Review of Recent Research in Treatment Approaches of Mucopolysaccharidosis (MPS)
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Yang A-Ram
Kim Jin-Sup
Cho Sung-Yoon
Jin Dong-Kyu
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Abstract
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Mucopolysaccharidosis (MPS) is caused by accumulation of the glycosaminoglycans in all tissues due to decreased activity of the lysosomal enzyme. Patients exhibit multisystemic signs and symptoms in a chronic and progressive manner, especially with changes in the skeleton, cardiopulmonary system, central nervous system, cornea, skin, liver, and spleen. In the past, treatment of MPS was limited to enzyme replacement therapy (ERT). The outcome for affected patients improved with the introduction of new technologies as hematopoietic stem cell transplantation, relegated to specific situations after ERT became available. Intrathecal ERT may be considered in situations of high neurosurgical risk but still it is experimental in humans. New insights on the pathophysiology of MPS disorders are leading to alternative therapeutic approaches, as gene therapy, inflammatory response modulators and substrate reduction therapy. In this paper, we will highlight the recent novel treatment and clinical trials for MPS and discuss with the goal of fostering an understanding of this field.
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KEYWORD
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MPS, Mucopolysaccharidosis, Clinical trials, Enzyme replacement therapy
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