New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
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New Zealand: Taylor & Francis Ltd
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Language
English
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Publisher
New Zealand: Taylor & Francis Ltd
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Niemann-Pick disease type C (NP-C) is an autosomal recessive disorder characterized by progressive neurological deterioration leading to premature death. The disease is caused by mutations in one of two genes, NPC1 or NPC2, leading to impaired intracellular lipid transport and build-up of lipids in various tissues, particularly the brain. Miglustat...
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New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
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TN_cdi_doaj_primary_oai_doaj_org_article_33acba7c06404c4685a8e9d10f38268e
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_33acba7c06404c4685a8e9d10f38268e
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ISSN
1178-203X,1176-6336
E-ISSN
1178-203X
DOI
10.2147/TCRM.S5777
