American journal of respiratory and critical care medicine
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Am. J. Respir. Crit. Care Med. · Jul 2014
Multicenter Study Observational StudyClinical Mechanism of the CFTR Potentiator Ivacaftor in G551D-Mediated Cystic Fibrosis.
Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator recently approved for patients with CF age 6 and older with the G551D mutation. ⋯ Significant clinical and physiologic improvements were observed on initiation of ivacaftor in a broad patient population, including reduced infection with P. aeruginosa. Biomarker studies substantially improve the understanding of the mechanistic consequences of CFTR modulation on pulmonary and gastrointestinal physiology.