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Discovery of GLPG2451, a Novel Once Daily Potentiator for the Treatment of Cystic Fibrosis

5 Jan 2021

Abstract excerpt

Cystic fibrosis (CF) is a life-threatening recessive genetic disease caused by mutations in the gene encoding for the cystic fibrosis transmembrane conductance regulator (CFTR). With the discovery of Ivacaftor and Lumacaftor, it has been shown that administration of one or more small molecules can partially restore the CFTR function. Correctors are small molecules that enhance the amount of CFTR on the cell...

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Share to Advanced biosensing and bioanalysis techniques
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PMID: 33399458
DOI: 10.1021/acs.jmedchem.0c01796

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026.

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