Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study

KIWI Study Group, Jane C Davies, Steve Cunningham, William T Harris, Allen Lapey, Warren E Regelmann, Gregory S Sawicki, Kevin W Southern, Sarah Robertson, Yulia Green, Jon Cooke, Margaret Rosenfeld

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