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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
9
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pubmed:dateCreated |
2007-8-20
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pubmed:databankReference |
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pubmed:abstractText |
Niemann-Pick type C disease (NPC) is an inherited neurodegenerative disorder characterised by an intracellular lipid-trafficking defect with secondary accumulation of glycosphingolipids. Miglustat, a small iminosugar, reversibly inhibits glucosylceramide synthase, which catalyses the first committed step of glycosphingolipid synthesis. Miglustat is able to cross the blood-brain barrier, and is thus a potential therapy for neurological diseases. We aimed to establish the effect of miglustat on several markers of NPC severity.
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pubmed:commentsCorrections |
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
IM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Sep
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pubmed:issn |
1474-4422
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pubmed:author |
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pubmed:issnType |
Print
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pubmed:volume |
6
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
765-72
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pubmed:dateRevised |
2007-11-24
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pubmed:meshHeading |
pubmed-meshheading:17689147-1-Deoxynojirimycin,
pubmed-meshheading:17689147-Adolescent,
pubmed-meshheading:17689147-Adult,
pubmed-meshheading:17689147-Age Factors,
pubmed-meshheading:17689147-Analysis of Variance,
pubmed-meshheading:17689147-Child,
pubmed-meshheading:17689147-Child, Preschool,
pubmed-meshheading:17689147-Cohort Studies,
pubmed-meshheading:17689147-Deglutition,
pubmed-meshheading:17689147-Double-Blind Method,
pubmed-meshheading:17689147-Drug Administration Schedule,
pubmed-meshheading:17689147-Enzyme Inhibitors,
pubmed-meshheading:17689147-Female,
pubmed-meshheading:17689147-Humans,
pubmed-meshheading:17689147-Male,
pubmed-meshheading:17689147-Niemann-Pick Disease, Type C,
pubmed-meshheading:17689147-Retrospective Studies,
pubmed-meshheading:17689147-Saccades,
pubmed-meshheading:17689147-Severity of Illness Index
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pubmed:year |
2007
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pubmed:articleTitle |
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study.
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pubmed:affiliation |
Department of Neurology and Pediatrics, Columbia University, NY, USA. patterson.marc@mayo.edu
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pubmed:publicationType |
Journal Article,
Clinical Trial,
Randomized Controlled Trial,
Research Support, Non-U.S. Gov't
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