Title : Clinical efficacy of enzyme replacement therapy in paediatric Hunter patients, an independent study of 3.5 years.

Pub. Date : 2014 Sep 18

PMID : 25231261






1 Functional Relationships(s)
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1 BACKGROUND: Hunter Syndrome is an X-linked lysosomal storage disorder due to the deficit of iduronate 2-sulfatase, an enzyme catalysing the degradation of the glycosaminoglycans (GAG) dermatan- and heparan-sulfate. Glycosaminoglycans iduronate 2-sulfatase Homo sapiens