A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants

A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants

Trikafta, a triple-combination drug, consisting of folding correctors VX-661 (tezacaftor), VX-445 (elexacaftor) and the gating potentiator VX-770 (ivacaftor) provided unprecedented clinical benefits for patients with the most common cystic fibrosis (CF) mutation, F508del. Trikafta indications were r...

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Bibliographic Details
Main Authors:	Guido Veit, Tony Velkov, Haijin Xu, Nathalie Vadeboncoeur, Lara Bilodeau, Elias Matouk, Gergely L. Lukacs
Format:	Article
Language:	English
Published:	MDPI AG 2021-07-01
Series:	Journal of Personalized Medicine
Subjects:	cystic fibrosis cystic fibrosis transmembrane conducatance regulator (CFTR) CFTR modulator combination primary human nasal epithelia CFTR missense mutations precision medicine
Online Access:	https://www.mdpi.com/2075-4426/11/7/643

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