Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
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Publisher
England: BioMed Central Ltd
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Language
English
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Publisher
England: BioMed Central Ltd
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Contents
Hurler syndrome (MPS IH), the severe, neurodegenerative form of type one mucopolysaccharidosis, is associated with rapid neurocognitive decline during toddlerhood and multi-system dysfunction. It is now standardly treated with hematopoietic cell transplantation (HCT), which halts accumulating disease pathology and prevents early death. While norm-b...
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Full title
Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
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TN_cdi_doaj_primary_oai_doaj_org_article_bbaed3c8c6b842ffb7a9f6ddf6649ef6
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_bbaed3c8c6b842ffb7a9f6ddf6649ef6
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-018-0817-3
