Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome)
Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome)
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New Zealand: Dove Medical Press Limited
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Language
English
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Publisher
New Zealand: Dove Medical Press Limited
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Contents
Mucopolysaccharidosis VII (MPS VII, Sly syndrome) is an ultra-rare lysosomal disease caused by a deficiency of the enzyme β-glucuronidase (GUS). The diagnosis is suspected based on a range of symptoms that are common to many other MPS types, and it is confirmed through biochemical and molecular studies. Besides supportive treatment, current and eme...
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Full title
Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome)
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TN_cdi_doaj_primary_oai_doaj_org_article_13d9067cc0774204885606dd2625edaf
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_13d9067cc0774204885606dd2625edaf
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ISSN
1176-6336,1178-203X
E-ISSN
1178-203X
DOI
10.2147/TCRM.S351300
