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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Detalles Bibliográficos
Autores principales: Matsuo, Muneaki, Shraishi, Koki, Wada, Koki, Ishitsuka, Yoichi, Doi, Hirohito, Maeda, Miyuki, Mizoguchi, Tatsuhiro, Eto, Junya, Mochinaga, Sakiko, Arima, Hidetoshi, Irie, Tetsumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2014
Materias:
The Lysosome
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5121362/
https://www.ncbi.nlm.nih.gov/pubmed/27896112
http://dx.doi.org/10.1016/j.ymgmr.2014.08.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5121362/
https://www.ncbi.nlm.nih.gov/pubmed/27896112
http://dx.doi.org/10.1016/j.ymgmr.2014.08.004

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