Title : [Clinical study of enzyme replacement therapy with idursulfase].

Pub. Date : 2007 Feb 19

PMID : 17345556






2 Functional Relationships(s)
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1 DEVELOPMENT: A preclinical trial in a knockout mouse showed a decrease in glycosaminoglycans, both in urine and in tissues, following treatment with idursulfase. Glycosaminoglycans iduronate 2-sulfatase Homo sapiens
2 In a randomised, double-blind, placebo-controlled clinical study in phase I/II conducted in 12 patients with Hunter syndrome, treatment with idursulfase displayed a good safety profile and also a decrease in the excretion of glycosaminoglycans and cases of visceromegaly. Glycosaminoglycans iduronate 2-sulfatase Homo sapiens