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In vivo Efficacy and Safety Evaluation of Lactosyl-β-cyclodextrin as a Therapeutic Agent for Hepatomegaly in Niemann-Pick Type C Disease

Niemann-Pick type C disease (NPC) is a fatal, autosomal recessive disorder, which causes excessive accumulation of free cholesterol in endolysosomes, resulting in progressive hepatomegaly and neurodegeneration. Currently, 2-hydroxypropyl-β-cyclodextrin (HP-β-CyD) is used at a high dose for the treat...

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Detalles Bibliográficos
Autores principales:	Maeda, Yuki, Motoyama, Keiichi, Nishiyama, Rena, Higashi, Taishi, Onodera, Risako, Nakamura, Hideaki, Takeo, Toru, Nakagata, Naomi, Yamada, Yusei, Ishitsuka, Yoichi, Kondo, Yuki, Irie, Tetsumi, Era, Takumi, Arima, Hidetoshi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6566927/ https://www.ncbi.nlm.nih.gov/pubmed/31130658 http://dx.doi.org/10.3390/nano9050802

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