Abstract
Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent US Food and Drug Administration (FDA) approval of lumacaftor combined with ivacaftor (Orkambi) targets patients with the F508del-CFTR. The question remains: Is this breakthrough combination therapy the “magic-bullet” cure for the vast majority of patients with CF? This review covers the contemporary clinical and scientific knowledge-base for lumacaftor/ivacaftor and highlights the emerging issues from recent conflicting literature reports.
Original language | English |
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Pages (from-to) | 130-141 |
Number of pages | 12 |
Journal | Clinical Pharmacology & Therapeutics |
Volume | 101 |
Issue number | 1 |
DOIs | |
Publication status | Published - 1 Jan 2017 |