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Lookup NU author(s): Dr Iram Haq, Dr Noreen Zainal Abidin, Attaybenes Socas Rodríguez, Dr Andrea Gonzalez-Ciscar, Dr Aaron Ions GardnerORCiD, Dr Malcolm Brodlie
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© 2019 Elsevier Ltd Cystic fibrosis (CF) is a life-limiting genetic disease that arises from defects in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and protein. This causes abnormal epithelial ion transport. CF is a multisystem condition but airway infection and inflammation carry the greatest treatment burden and are the predominant cause of morbidity and mortality. Novel therapeutic strategies have recently been developed to target specific molecular defects arising from CFTR mutations. These CFTR modulator therapies are designed to correct the underlying protein processing defect or to potentiate CFTR function to improve ion transport. Such advances have revolutionized CF treatment approaches, which have previously only addressed downstream effects, and hold significant promise for the future. This review will summarize recent advances in CFTR modulator development and how these have impacted upon treatment outcomes and the lives of people with CF.
Author(s): Haq IJ, Parameswaran MC, Abidin NZ, Socas A, Gonzalez-Ciscar A, Gardner AI, Brodlie M
Publication type: Review
Publication status: Published
Journal: Paediatrics and Child Health
Year: 2019
Volume: 29
Issue: 4
Pages: 151-157
Print publication date: 01/04/2019
Online publication date: 13/02/2019
Acceptance date: 02/04/2018
ISSN (print): 1751-7222
ISSN (electronic): 1878-206X
Publisher: Churchill Livingstone
URL: https://doi.org/10.1016/j.paed.2019.01.011
DOI: 10.1016/j.paed.2019.01.011