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
Desnick, R. J. & Schuchman, E. H. Enzyme replacement and enhancement therapies: lessons from lysosomal disorders. Nature Rev. Genet. 3, 954–966 (2002).
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).
Anson, D. S. et al. Correction of human mucopolysaccharidosis type-VI fibroblasts with recombinant N-acetylgalactosamine-4-sulphatase. Biochem. J. 284, 789–794 (1992).
Crawley, A. C. et al. Enzyme replacement therapy in a feline model of Maroteaux–Lamy syndrome. J. Clin. Invest. 97, 1864–1873 (1996).
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).
FDA labelling information [online], <http://www.fda.gov/cder/foi/label/2005/021877lbl.pdf> (2005).
Harmatz, P. et al. Enzyme replacement therapy in mucopolysaccharidosis type VI (Maroteaux–Lamy syndrome). J. Pediatr. 144, 574–580 (2004).
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).
Meikle, P. J. et al. Newborn screening for lysosomal storage disorders: evaluation of protein profiling. J. Inherit. Met. Dis. 28, 14 (2005).
Li, Y. et al. (2004) Direct multiplex assay of lysosomal enzymes in dried blood spots for newborn screening. Clin. Chem. 50, 1785–1796 (2004).
Meikle, P. J., Hopwood, J. J. & Clague, A. E. Prevalence of lysosomal storage disorders. JAMA 281, 249–254 (1999).
Genzyme data <http://www.genzyme.com> (2006).
Shire data <http://www.shire.com> (2006).
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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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DOI: https://doi.org/10.1038/nrd1962
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