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Galsulfase

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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.

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References

  1. Desnick, R. J. & Schuchman, E. H. Enzyme replacement and enhancement therapies: lessons from lysosomal disorders. Nature Rev. Genet. 3, 954–966 (2002).

    Article  CAS  Google Scholar 

  2. Neufeld, E. F. & Muenzer, J. in The Metabolic and Molecular Bases of Inherited Disease (eds Scriver, C. et al.) 3421–3452 (McGraw-Hill, New York, 2001).

    Google Scholar 

  3. Anson, D. S. et al. Correction of human mucopolysaccharidosis type-VI fibroblasts with recombinant N-acetylgalactosamine-4-sulphatase. Biochem. J. 284, 789–794 (1992).

    Article  CAS  Google Scholar 

  4. Crawley, A. C. et al. Enzyme replacement therapy in a feline model of Maroteaux–Lamy syndrome. J. Clin. Invest. 97, 1864–1873 (1996).

    Article  CAS  Google Scholar 

  5. Crawley, A. C. et al. Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI. J. Clin. Invest. 99, 651–662 (1997).

    Article  CAS  Google Scholar 

  6. FDA labelling information [online], <http://www.fda.gov/cder/foi/label/2005/021877lbl.pdf> (2005).

  7. Harmatz, P. et al. Enzyme replacement therapy in mucopolysaccharidosis type VI (Maroteaux–Lamy syndrome). J. Pediatr. 144, 574–580 (2004).

    Article  CAS  Google Scholar 

  8. Kakkis, E. et al. Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. Mol. Genet. Metab. 83, 163–174 (2004).

    Article  CAS  Google Scholar 

  9. Meikle, P. J. et al. Newborn screening for lysosomal storage disorders: evaluation of protein profiling. J. Inherit. Met. Dis. 28, 14 (2005).

    Google Scholar 

  10. Li, Y. et al. (2004) Direct multiplex assay of lysosomal enzymes in dried blood spots for newborn screening. Clin. Chem. 50, 1785–1796 (2004).

    Article  CAS  Google Scholar 

  11. Meikle, P. J., Hopwood, J. J. & Clague, A. E. Prevalence of lysosomal storage disorders. JAMA 281, 249–254 (1999).

    Article  CAS  Google Scholar 

  12. Genzyme data <http://www.genzyme.com> (2006).

  13. Shire data <http://www.shire.com> (2006).

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Authors and Affiliations

  1. the Lysosomal Diseases Research Unit, Women's and Children's Hospital, 72 King William Road, North Adelaide, 5006, South Australia, Australia

    John J. Hopwood

  2. IMS Health, 7 Harewood Avenue, London, NW1 6JB, UK

    Guy Bate

  3. Nature Reviews Drug Discovery, UK

    Peter Kirkpatrick

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  1. John J. Hopwood
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  2. Guy Bate
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  3. Peter Kirkpatrick
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Hopwood, J., Bate, G. & Kirkpatrick, P. Galsulfase. Nat Rev Drug Discov 5, 101–102 (2006). https://doi.org/10.1038/nrd1962

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