Elexacaftor/tezacaftor/ivacaftor improves CFTR function to near-normal levels in children with cystic fibrosis.
BACKGROUND The CFTR modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI) improves CFTR function in adolescents and adults with cystic fibrosis (CF) carrying at least one F508del allele to around 40% of median CFTR activity in non-CF controls. This partial restoration of CFTR function is associated with residual muc...