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Modulator therapies for cystic fibrosis

Lookup NU author(s): Dr Iram HaqORCiD, 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.

Publication metadata

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


DOI: 10.1016/j.paed.2019.01.011