Affordable Access

deepdyve-link
Publisher Website

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

Authors
  • Middleton, Peter G1
  • Taylor-Cousar, Jennifer L2
  • 1 Cystic Fibrosis Unit, Ludwig Engel Centre for Respiratory Research, Westmead Millennium Institute, University of Sydney at Westmead, Westmead, Australia. , (Australia)
  • 2 National Jewish Health, Pulmonary, Internal Medicine and Pediatrics, Denver, CO, USA.
Type
Published Article
Journal
Expert Review of Respiratory Medicine
Publisher
Informa UK (Taylor & Francis)
Publication Date
Jun 01, 2021
Volume
15
Issue
6
Pages
723–735
Identifiers
DOI: 10.1080/17476348.2021.1855980
PMID: 33249928
Source
Medline
Keywords
Language
English
License
Unknown

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.

Report this publication

Statistics

Seen <100 times