Ivacaftor in cystic fibrosis with residual function: Lung function results from an N-of-1 study

Ivacaftor (Kalydeco ®) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that enhances chloride transport in multiple mutant CFTR forms in vitro, including the G551D-CFTR mutation, other severe gating mutations, and certain mutations associated with residual CFTR function [1–3]. Clinical st udies first established the efficacy and safety of ivacaftor in patients with CF and a G551D mutation or other severe gating mutations (ie, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R) [4–9].

Source: Journal of Cystic Fibrosis - November 25, 2019 Category: Respiratory Medicine Authors: Jerry A. Nick, Connie St. Clair, Marion C. Jones, Lan Lan, Mark Higgins, VX12-770-113 Study Team Tags: Original Article Source Type: research