These include the CFTR potentiator compound ivacaftor (VX-770) that can improve channel activity in CFTR gating mutations present in ∼5% of CF patients and the combination of ivacaftor with the CFTR corrector compound lumacaftor (VX-809) that has been approved for F508del homozygous patients (Ratjen et al., 2017). Here, CFTR is linked to cystic fibrosis.