Development of elexacaftor - tezacaftor - ivacaftor: Highly effective CFTR modulation for the majority of people with Cystic Fibrosis.
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| Abstract |
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. Survival has increased dramatically over the last 50 years, from 10 years in the 1960s to 30 years in the 1990s and approximately 50 years currently.Areas covered: This review will examine the development of highly effective modulators for CF which will revolutionize therapy for more than 90% of the people with CF. This review summarizes the development of triple combination CFTR modulator elexacaftor-tezacaftor-ivacaftor.Expert opinion: The development of this highly effective CFTR modulator for the majority of people with CF will likely change the landscape of CF care. The challenge is to now find highly effective therapy for the remaining 10% of the people with CF who may need other therapeutic agents to correct their primary defect.
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| Authors | Peter G Middleton, Jennifer L Taylor-Cousar |
| Journal | Expert review of respiratory medicine (Expert Rev Respir Med) Vol. 15 Issue 6 Pg. 723-735 (06 2021) ISSN: 1747-6356 [Electronic] England |
| PMID | 33249928 (Publication Type: Journal Article, Review) |
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