CFTR Modulator Therapies for Cystic Fibrosis

The cloning of cystic fibrosis transmembrane conductance regulator (CFTR) set into motion a cascade of discoveries that have helped to reveal the underlying pathophysiologic basis of cystic fibrosis (CF). This discovery and the knowledge that followed have also provided the opportunity to target this basic defect, with the hope of reversing or preventing the serious clinical consequences that result from absent CFTR function. With the recent approval of 2 therapies that directly modulate CFTR function in more than half of the CF population, we are now at the beginning of a pathway to providing increasingly effective therapies that have the potential to provide a fundamental change in the outcome of most patients with CF.