Efficacy and safety of ivacaftor treatment: randomized trial in subjects with cystic fibrosis who have an R117H-CFTR mutation

摘要

BackgroundIvacaftor, approved for treatment of cystic fibrosis (CF) patients aged ≥6 years with G551D-CFTR or other gating mutations, was evaluated in subjects with R117H-CFTR, a residual function mutation.