Clinical and functional efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis carrying the N1303K mutation

•Recent in vitro data suggested that ELX/TEZ/IVA increases N1303K-CFTR function.•8 pwCF that started off label ELX/TEZ/IVA and had significant clinical response, with increase in FEV1 and BMI, though sweat chloride remained in the pathologic value.•Although not listed as ELX/TEZ/IVA responsive mutatoin, PwCF carrying the N1303K mutation may have significant clinical benefit by this treatment.