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100. A multicentre retrospective survey of miglustat in patients with Niemann-Pick type C disease

✍ Scribed by Marc Patterson; Merci Pineda; Fridiric Sedel; Eugen Mengel; Wuh-Liang Hwu; Marianne Rohrbach; Brubo Bembi; Christoph Korenke; Cicile Luzy; Peter Schieber; Ruben Giorgino; Ed Wraith

Book ID: 116988534
Publisher: Elsevier Science
Year: 2009
Tongue: English
Weight: 43 KB
Volume: 96
Category: Article
ISSN: 1096-7192
DOI: 10.1016/j.ymgme.2008.11.101

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## Abstract Niemann–Pick disease type C (NPC) is a progressive neurodegenerative disorder for which there is no effective treatment other than supportive therapy. Recently, the oral medication miglustat has been offered as a possible therapy aimed at reducing pathological substrate accumulation. Th