Idursulfase for the treatment of mucopolysaccharidosis II.

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Clarke LA

Idursulfase for the treatment of mucopolysaccharidosis II.

Expert Opin Pharmacother. 2008 Feb;9(2):311-7. doi: 10.1517/14656566.9.2.311 .

PubMed ID
18201153 [ View in PubMed
]
Abstract

Human recombinant proteins are being used to treat an increasing number of disorders. Advances in the large scale production of recombinant proteins and the understanding of glycosylation and its importance for protein targeting and function have led to the development of recombinant enzyme-replacement regimens for a number of human lysosomal storage diseases. The latest addition to this group is mucopolysaccharidosis II or Hunter syndrome. Purified human recombinant idursulfase has been shown to alter disease manifestations in individuals with Hunter syndrome. The recent approval in the US, Europe, Canada and Japan of idursulfase for the treatment of Hunter syndrome introduces the first pharmacologic agent and indeed the first specific treatment directed towards this devastating genetic disease.

DrugBank Data that Cites this Article

Drugs
Drug Targets
DrugTargetKindOrganismPharmacological ActionActions
IdursulfasePerilipin-3ProteinHumans
No
Not AvailableDetails