Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann–Pick Type C disease

Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann–Pick Type C disease

Niemann–Pick Type C disease (NPC) is an autosomal recessive lysosomal storage disorder characterized by progressive neurological deterioration. Previously, we reported that intravenous administration of 2-hydroxypropyl-β-cyclodextrin (HPB-CD) in two patients with NPC had only partial and transient b...

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Bibliographic Details
Main Authors: Muneaki Matsuo, Koki Shraishi, Koki Wada, Yoichi Ishitsuka, Hirohito Doi, Miyuki Maeda, Tatsuhiro Mizoguchi, Junya Eto, Sakiko Mochinaga, Hidetoshi Arima, Tetsumi Irie
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Niemann–Pick Type C disease
Cyclodextrin
Cholesterol
Intrathecal administration
Intracerebroventricular administration
Pharmacokinetics
Online Access:http://www.sciencedirect.com/science/article/pii/S221442691400055X

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http://www.sciencedirect.com/science/article/pii/S221442691400055X

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