Modulators of CFTR. Updates on clinical development and future directions

Emmanuelle E Bardin, Alexandra Pastor, Michaela Semeraro, Anita Golec, Kate Hayes, Benoit Chevalier, Farouk Berhal, Guillaume Prestat, Alexandre Hintzpeter, Christine Gravier-Pelletier, Iwona Pranke, Isabelle Sermet-Gaudelus*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

10 Citations (Scopus)
28 Downloads (Pure)


Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Current symptomatic CF therapies, which treat the downstream consequences of of CFTR mutations, have increased survival. Better knowledge of the of the CFTR protein has enabled pharmacologic therapy aiming to restore mutated CFTR expression and function. These CFTR "modulators" have revolutionised the CF therapeutic landscape, with the potential to transform prognosis for a considerable number of patients. This review provides a brief summary of their mechanism of action and presents a thorough review of the results obtained from clinical trials of CFTR modulators.
Original languageEnglish
Article number113195
Number of pages15
JournalEuropean journal of medicinal chemistry
Early online date16 Jan 2021
Publication statusPublished - Mar 2021
Externally publishedYes


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