Intracerebroventricular 2-hydroxypropyl-β-cyclodextrin improves not only neurological symptoms but also hepatic abnormalities in Niemann-Pick disease type C model mice and patients

Name: Work Video:
Duration: 3 min 30 s
Madoka Fukaura; Yoichi Ishituka; Yuki Kondo; Ushihama Naoki; Toru Takeo; Naomi Nakagata; Takumi Era; Taishi Higashi; Keiichi Motoyama; Hidetoshi Arima; Yuki Kurauchi; Hiroshi Katsuki; Shunsuke Kamei; Tsuyoshi Shuto; Hirofumi Kai; Katsumi Higaki; Yoshio Sakiyama; Takafumi Sakakibara; Muneaki Matsuo; Tetsumi Irie
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