Functional rescue of an ABCB11 mutant by ivacaftor: A new targeted pharmacotherapy approach in bile salt export pump deficiency |
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| Authors: | Elodie Mareux,Martine Lapalus,Rachida Amzal,Marion Almes,Tounsia Aï t‐Slimane,Jean‐Louis Delaunay,Pauline Adnot,Mauricette Collado‐Hilly,Anne Davit‐Spraul,Thomas Falgui res,Isabelle Callebaut,Emmanuel Gonzales,Emmanuel Jacquemin |
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| Affiliation: | Elodie Mareux,Martine Lapalus,Rachida Amzal,Marion Almes,Tounsia Aït‐Slimane,Jean‐Louis Delaunay,Pauline Adnot,Mauricette Collado‐Hilly,Anne Davit‐Spraul,Thomas Falguières,Isabelle Callebaut,Emmanuel Gonzales,Emmanuel Jacquemin |
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| Abstract: | |
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| Keywords: | ABC transporters superfamily cholestatic liver diseases paediatrics PFIC2 potentiator VX‐770 |
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