• Pediatr. Clin. North Am. · Aug 2016

    Review

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

    • Thida Ong and Bonnie W Ramsey.
    • Division of Pulmonary and Sleep Medicine, Department of Pediatrics, Seattle Children's Hospital, University of Washington, 4800 Sand Point Way Northeast, M/S OC.7.720, Seattle, WA 98105, USA.
    • Pediatr. Clin. North Am. 2016 Aug 1; 63 (4): 751-64.

    AbstractCystic 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 population responsive to this therapy and future approaches to predict and monitor treatment response of CFTR modulators are discussed. The goal remains to expand patient-centered and personalized therapy to all patients with CF, ultimately improving life expectancy and quality of life for this disease.Copyright © 2016 Elsevier Inc. All rights reserved.

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