Article
Effect of food on the pharmacokinetics and therapeutic efficacy of 4-phenylbutyrate in progressive familial intrahepatic cholestasis.
Scientific reports - 19 Nov 2019
Nakano Satoshi, Osaka Shuhei, Sabu Yusuke, Minowa Kei, Hirai Saeko, Kondou Hiroki, Kimura Takeshi, Azuma Yoshihiro, Watanabe Satoshi, Inui Ayano, Bessho Kazuhiko, Nakamura Hidefumi, Kusano Hironori, Nakazawa Atsuko, Tanikawa Ken, Kage Masayoshi, Shimizu Toshiaki, Kusuhara Hiroyuki, Zen Yoh, Suzuki Mitsuyoshi, Hayashi Hisamitsu
Abstract excerpt
Progressive familial intrahepatic cholestasis (PFIC), a rare inherited disorder, progresses to liver failure in childhood. We have shown that sodium 4-phenylbutyrate (NaPB), a drug approved for urea cycle disorders (UCDs), has beneficial effects in PFIC. However, there is little evidence to determine an optimal regimen for NaPB therapy. Herein, a multicenter, open-label, single-dose study was performed to...
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