Longitudinal changes in cerebellar and subcortical volumes in adult-onset Niemann-Pick disease type C patients treated with miglustat

Elizabeth Bowman, Mark Walterfang, Larry Allen Abel, Patricia M Desmond, Michael Collingwood Fahey, Dennis Velakoulis

Research output: Contribution to journalArticleResearchpeer-review

Abstract

Niemann-Pick disease type C (NPC) is a rare neurovisceral disorder resulting in impaired intracellular lipid trafficking. The only disease-modifying treatment available to date is miglustat, an iminosugar inhibiting the accumulation of lipid by-products in neurons. This study explored how changes in cerebellar grey and white matter volumes, and in subcortical volumes, related to patient treatment status and disability and ataxia ratings.
Original languageEnglish
Pages (from-to)2106 - 2114
Number of pages9
JournalJournal of Neurology
Volume262
Issue number9
DOIs
Publication statusPublished - 2015

Cite this

Bowman, Elizabeth ; Walterfang, Mark ; Abel, Larry Allen ; Desmond, Patricia M ; Fahey, Michael Collingwood ; Velakoulis, Dennis. / Longitudinal changes in cerebellar and subcortical volumes in adult-onset Niemann-Pick disease type C patients treated with miglustat. In: Journal of Neurology. 2015 ; Vol. 262, No. 9. pp. 2106 - 2114.
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Longitudinal changes in cerebellar and subcortical volumes in adult-onset Niemann-Pick disease type C patients treated with miglustat. / Bowman, Elizabeth; Walterfang, Mark; Abel, Larry Allen; Desmond, Patricia M; Fahey, Michael Collingwood; Velakoulis, Dennis.

In: Journal of Neurology, Vol. 262, No. 9, 2015, p. 2106 - 2114.

Research output: Contribution to journalArticleResearchpeer-review

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AU - Abel, Larry Allen

AU - Desmond, Patricia M

AU - Fahey, Michael Collingwood

AU - Velakoulis, Dennis

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