Treatment with miglustat reverses the lipid-trafficking defect in Niemann–Pick disease type C

Niemann–Pick disease type C (NP-C) is a hereditary neurovisceral lipid storage disorder. Although traditionally considered a primary cholesterol storage disorder, a variety of glycolipids accumulate in NP-C cells, which resemble those from glycosphingolipidosis patients. Substrate reduction therapy...

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Bibliographic Details

Published in:	Neurobiology of Disease
Main Authors:	Robin H Lachmann, Danielle te Vruchte, Emyr Lloyd-Evans, Gabriele Reinkensmeier, Daniel J Sillence, Luisa Fernandez-Guillen, Raymond A Dwek, Terry D Butters, Timothy M Cox, Frances M Platt
Format:	Article
Language:	English
Published:	Elsevier 2004-08-01
Subjects:	Niemann–Pick disease type C Substrate reduction therapy Miglustat Glycosphingolipidoses Lipid trafficking
Online Access:	http://www.sciencedirect.com/science/article/pii/S0969996104001111