机译:ivacaftor 对 ABCB11 突变体的功能性挽救:一种新的靶向药物治疗方法治疗胆盐输出泵缺乏症
Univ Paris Saclay, UMR S 1193, Physiopathogenese & Traitement Malad Foie, Inserm,Hepatinov, Orsay;
Sorbonne Univ, IMPMC, Inst Mineral Phys Mat & Cosmochim, Museum Natl Hist Nat,UMR CNRS 7590, Paris;
Sorbonne Univ, Inst Cardiometab & Nutr ICAN, UMR S 938, Inserm,Ctr Rech St Antoine CRSA, Paris;
ABC transporters superfamily; cholestatic liver diseases; paediatrics; PFIC2; potentiator; VX-770;