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Article

New Therapeutic Approaches to Modulate and Correct Cystic Fibrosis Transmembrane Conductance Regulator.

Pediatric clinics of North America - 1 Aug 2016

Ong Thida, Ramsey Bonnie W

Abstract excerpt

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are clinically available personalized medicines approved for some individuals with cystic fibrosis (CF) to target the underlying defect of disease. This review summarizes strategies used to develop CFTR modulators as therapies that improve function and availability of CFTR protein. Lessons learned from dissemination of ivacaftor across the CF...

Topics

Aminophenols
Aminopyridines
Benzodioxoles
Chloride Channel Agonists
Cystic Fibrosis
Cystic Fibrosis Transmembrane Conductance Regulator
Humans
Molecular Targeted Therapy
Mutation
Quinolones

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 27469186
DOI: 10.1016/j.pcl.2016.04.006
PMCID: PMC5478192

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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