Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

Cystic fibrosis is a progressive, systemic, life-limiting, autosomal recessive disease that is caused by reduced quantity or function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein due to mutations in the CFTR gene. Loss of chloride transport activity due to defects in…

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