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: Matsuo, Muneaki, Shraishi, Koki, Wada, Koki, Ishitsuka, Yoichi, Doi, Hirohito, Maeda, Miyuki, Mizoguchi, Tatsuhiro, Eto, Junya, Mochinaga, Sakiko, Arima, Hidetoshi, Irie, Tetsumi
Format: Online
Language:English
Published: Elsevier 2014
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5121362/

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5121362/

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