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|Scopus||Web of Science®||Altmetric|
|Citation:||Nature Reviews Drug Discovery, 2006; 5(2):101-102|
|Publisher:||Nature Publishing Group|
|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.|
|Appears in Collections:||Aurora harvest|
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