Keywords

CF, CFTR gene, CFTR modulators, cystic fibrosis, cystic fibrosis transmembrane conductance regulator gene

Authors

1. Wendekier, Camille PhD, CNE, RN
2. Wendekier-Raybuck, Katheryn PharmD

Abstract

Abstract: Cystic fibrosis is an autosomal recessive genetic disorder that causes a lifetime of debilitating and potentially fatal complications affecting the lungs and other organ systems. Over 1,700 gene mutations that cause this rare disorder have been identified. This article describes the current treatment landscape for adults with CF, including the 2019 FDA approval of a breakthrough triple-drug combination therapy that may significantly improve the quality of life for an estimated 90% of patients with CF.