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Development of elexacaftor - tezacaftor - ivacaftor: Highly effective CFTR modulation for the majority of people with Cystic Fibrosis.

Expert review of respiratory medicine - 1 Jun 2021

Middleton Peter G, Taylor-Cousar Jennifer L

Abstract excerpt

Introduction: Cystic fibrosis (CF), the most common life-shortening inherited disorder in people of European descent, also occurs in other ethnicities. The identification of the disease, the isolation of the causative gene, termed the cystic fibrosis transmembrane conductance regulator (CFTR) and the improved survival from comprehensive multidisciplinary treatment is one of the success stories of modern medicine....

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PMID: 33249928
DOI: 10.1080/17476348.2021.1855980

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