Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two<i>F508del</i>alleles

Abstract excerpt

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene ( CFTR ) that result in diminished quantity and/or function of the CFTR anion channel. F508del-CFTR , the most common CF-causing mutation (found in ∼90% of patients), causes severe processing and trafficking defects, resulting in decreased CFTR quantity and function. CFTR modulators are medications that increase the...

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