Adelaide Research & Scholarship
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https://hdl.handle.net/2440/35766
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| Type: | Journal article |
| Title: | Galsulfase |
| Author: | Hopwood, J. Bate, G. Kirkpatrick, P. |
| Citation: | Nature Reviews Drug Discovery, 2006; 5(2):101-102 |
| Publisher: | Nature Publishing Group |
| Issue Date: | 2006 |
| ISSN: | 1474-1776 1474-1784 |
| Statement of Responsibility: | John J. Hopwood; Guy Bate; Peter Kirkpatrick |
| Abstract: | In 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. |
| Keywords: | CHO Cells Animals Humans Mucopolysaccharidosis VI N-Acetylgalactosamine-4-Sulfatase Recombinant Proteins Female Cricetinae |
| DOI: | 10.1038/nrd1962 |
| Published version: | http://dx.doi.org/10.1038/nrd1962 |
| Appears in Collections: | Aurora harvest Paediatrics publications |
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