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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Detalhes bibliográficos
Publicado no:Nanomaterials (Basel)
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: MDPI 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6566927/
https://ncbi.nlm.nih.gov/pubmed/31130658
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/nano9050802
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