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The story of cystic fibrosis 1965–2015
Author(s) -
Massie John,
Robinson Phillip J,
Cooper Peter J
Publication year - 2016
Publication title -
journal of paediatrics and child health
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.631
H-Index - 76
eISSN - 1440-1754
pISSN - 1034-4810
DOI - 10.1111/jpc.13309
Subject(s) - medicine , cystic fibrosis , cystic fibrosis transmembrane conductance regulator , ivacaftor , regulator , genetic enhancement , tobramycin , transmembrane protein , mutation , intensive care medicine , recombinant dna , immunology , gene , bioinformatics , pseudomonas aeruginosa , genetics , bacteria , biology , receptor
There have been dramatic changes in the care of children with cystic fibrosis ( CF ) from 1965 to 2015. The initial improvements were the result of incremental gains in medical knowledge and better use of available therapies. Some CF ‐specific drugs were developed (recombinant human DNase and purified tobramycin for inhalation), but these simply delayed the progression of the lung damage. The discovery of the CF transmembrane conductance regulator gene in 1989 created an explosion of knowledge of airway biology and CF pathophysiology. Now mutation‐specific therapy to potentiate mutated CF transmembrane conductance regulator is available for some patients with CF and is transforming their lives. Further advances are eagerly anticipated to benefit all patients with CF .