Assessment

tezacaftor/ivacaftor

Cystic fibrosis in patients aged 6 to 11 years who are heterozygous for the F508del mutation and carry one of the following CFTR mutations: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G and 3849+10kbC→T (residual function mutations)