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J Paediatr Child Health. 2016 Nov;52(11):991-994. doi: 10.1111/jpc.13309. Epub 2016 Sep 1.

The story of cystic fibrosis 1965-2015.

Author information

1
Department of Respiratory and Sleep Medicine, Royal Children's Hospital, Melbourne, Victoria, Australia.
2
Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia.
3
Infection, Immunity and Environment Theme, Murdoch Children's Research Institute, Melbourne, Victoria, Australia.
4
Department of Respiratory and Sleep Medicine, Children's Hospital at Westmead, Sydney, New South Wales, Australia.

Abstract

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.

KEYWORDS:

cystic fibrosis; history; review

PMID:
27586437
DOI:
10.1111/jpc.13309

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