Case report of the perinatal form of Nieman-Pick disease type С against the backdrop of pathogenetic off-label therapy by Nailya R. Mingacheva, Aelita A. Kamalova, Nargis A. Artykova, Razilya F. Rakhmaeva, Gulnara I. Sageeva

Subjects: Get full text

Inability of α-cyclodextrins to accommodate cholesterol potentially underlies their lack of efficacy and ototoxicity in Niemann-Pick disease type C treatment by Yusei Yamada, Mayuko Tanaka, Yusei Ikeda, Yuki Kondo, Toru Takeo, Naomi Nakagata, Toru Miwa, Hiroki Takeda, Yorihisa Orita, Keiichi Motoyama, Taishi Higashi, Hidetoshi Arima, Takahiro Seki, Yuki Kurauchi, Hiroshi Katsuki, Katsumi Higaki, Kotaro Matsusaka, Kentaro Minami, Naoki Yoshikawa, Ryuji Ikeda, Muneaki Matsuo, Tetsumi Irie, Yoichi Ishitsuka

Subjects: “…Niemann-Pick disease type C…”
Get full text