S18 Ivacaftor treatment in preschool children with cystic fibrosis and a CFTR gating mutation: extended evaluation

Abstract

Introduction and objectivesIvacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, has demonstrated improved outcomes in patients aged ≥6 years with cystic fibrosis (CF) and a CFTR gating mutation. The...