Possible phenylacetate hepatotoxicity during 4-phenylbutyrate therapy of Byler disease

BL Shneider, A Morris, J Vockley - Journal of pediatric …, 2016 - journals.lww.com
In vitro studies have suggested that 4-phenylbutyrate (PBA) may rescue missense mutated
proteins that underlie some forms of progressive familial intrahepatic cholestasis …