Mucopolysaccharidoses
Mucopolysaccharidoses therapy achieved by gene expression-targeted therapy
Published in Drug Week, September 8th, 2006
"MPS are inherited, severe, progressive, metabolic disorders caused by deficiencies in different enzymes involved in degradation of GAGs. Although enzyme replacement therapy (ERT) has recently been available for MPS type I, and clinical trials have been performed in ERT for MPS II and MPS VI, there is little chance that this kind of treatment may be effective for neurodegenerative forms of MPS (due to inefficient delivery of enzymes to central nervous system through the...
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Source: Drug Week (2006-09-08)
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