Efficacy of 2-Hydroxypropyl-β-cyclodextrin in Niemann–Pick Disease Type C Model Mice and Its Pharmacokinetic Analysis in a Patient with the Disease

Tanaka, Yuta ; Yamada, Yusei ; Ishitsuka, Yoichi ; Matsuo, Muneaki ; Shiraishi, Koki ; Wada, Koki ; Uchio, Yushiro ; Kondo, Yuki ; Takeo, Toru ; Nakagata, Naomi ; Higashi, Taishi ; Motoyama, Keiichi ; Arima, Hidetoshi ; Mochinaga, Sakiko ; Higaki, Katsumi ; Ohno, Kousaku ; Irie, Tetsumi

Biological and Pharmaceutical Bulletin, 2015/06/01, Vol.38(6), pp.844-851 [Periódico revisado por pares]

Japan: The Pharmaceutical Society of Japan

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