Effects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I
Effects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I
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United States: Public Library of Science
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Language
English
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Publisher
United States: Public Library of Science
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Contents
Mucopolysaccharidosis type I (MPS I) is a progressive disorder caused by deficiency of α-L-iduronidase (IDUA), which leads to storage of heparan and dermatan sulphate. It is suggested that early enzyme replacement therapy (ERT) leads to better outcomes, although many patients are diagnosed late and don't receive immediate treatment. This study aims...
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Full title
Effects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I
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TN_cdi_plos_journals_1650969676
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_1650969676
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ISSN
1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0117271
