Please use this identifier to cite or link to this item: https://hdl.handle.net/2440/35766
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dc.contributor.authorHopwood, J.-
dc.contributor.authorBate, G.-
dc.contributor.authorKirkpatrick, P.-
dc.date.issued2006-
dc.identifier.citationNature Reviews Drug Discovery, 2006; 5(2):101-102-
dc.identifier.issn1474-1776-
dc.identifier.issn1474-1784-
dc.identifier.urihttp://hdl.handle.net/2440/35766-
dc.description.abstractIn May 2005, galsulfase (Naglazyme; BioMarin), a recombinant form of human N-acetylgalactosamine 4-sulfatase, was approved by the US FDA for the treatment of patients with mucopolysaccharidosis type VI, a rare lysosomal storage disorder caused by a deficiency of N–acetylgalactosamine 4-sulfatase. It is the first approved product for the treatment of mucopolysaccharidosis type VI, and has been granted orphan drug status.-
dc.description.statementofresponsibilityJohn J. Hopwood; Guy Bate; Peter Kirkpatrick-
dc.language.isoen-
dc.publisherNature Publishing Group-
dc.subjectCHO Cells-
dc.subjectAnimals-
dc.subjectHumans-
dc.subjectMucopolysaccharidosis VI-
dc.subjectN-Acetylgalactosamine-4-Sulfatase-
dc.subjectRecombinant Proteins-
dc.subjectFemale-
dc.subjectCricetinae-
dc.titleGalsulfase-
dc.typeJournal article-
dc.identifier.doi10.1038/nrd1962-
pubs.publication-statusPublished-
Appears in Collections:Aurora harvest
Paediatrics publications

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