{"id":27,"date":"2024-11-18T12:13:41","date_gmt":"2024-11-18T12:13:41","guid":{"rendered":"https:\/\/www.naglazyme.com\/en-us\/?page_id=27"},"modified":"2026-05-11T08:04:08","modified_gmt":"2026-05-11T08:04:08","slug":"what-happens-in-mps-vi","status":"publish","type":"page","link":"https:\/\/www.naglazyme.com\/en-us\/what-happens-in-mps-vi\/","title":{"rendered":"What happens in MPS VI?"},"content":{"rendered":"
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What happens in MPS VI?<\/h1>\n

A person with MPS VI lacks an enzyme the body needs to get rid of waste in the cells1,2<\/sup><\/h2>\n

This waste is a jelly\u2010like product called GAG, which is short for \u201cglycosaminoglycan\u201d (GLY\u2010koh\u2010sah\u2010meen\u2010o\u2010GLY\u2010can).3<\/sup><\/p>\n

Over time, GAGs build up inside the cells, making them swell. Packed with GAGs, the cells don\u2019t work right. Since many body tissues and organs are made of the cells, they don\u2019t work right either. GAGs are stored in most cells of the body, so many major organs and systems can be harmed<\/a>. Most of the time, however, intelligence is not affected.4<\/sup><\/p>\n

NAGLAZYME\u00ae<\/sup> (galsulfase) provides the enzyme that the body needs to break down and get rid of GAGs to allow cells to work properly. In timed tests, people who took NAGLAZYME were able to\u00a0climb more stairs and walk farther than before<\/a>.5<\/sup><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Left: A healthy cell with just a tiny amount of GAGs. Right: An MPS cell with excessive buildup of GAGs.<\/em><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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References:<\/strong>\n<\/h4>\n\t\t\t\t\t\t
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  1. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, et al., eds. The Metabolic and Molecular Bases of Inherited Disease.<\/em> 8th ed. New York, NY: McGraw-Hill; 2001:3421-3452.\n<\/span><\/li>\n
  2. Coutinho MF, Lacerda L, Alves S. Glycosaminoglycan storage disorders: a review. Biochem Res Int.<\/em> 2012;2012:471325.\n<\/span><\/li>\n
  3. Swiedler SJ, Beck M, Bajbouj M, et al. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Am J Med Genet A.<\/em> 2005;134A(2):144-151.\n<\/span><\/li>\n
  4. Giugliani R, Lampe C, Guffon N, et al. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome): 10-year follow-up of patients previously participating in an MPS VI survey study. Am J Med Genet A.<\/em> 2014;164A(8):1953-1964.\n<\/span><\/li>\n
  5. NAGLAZYME [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.\n<\/span><\/li>\n <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>\n\n
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