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Drug combination that corrects deficient protein in cystic fibrosis improves lung function

BMJ 2015; 350 doi: https://doi.org/10.1136/bmj.h2689 (Published 19 May 2015) Cite this as: BMJ 2015;350:h2689
  1. Susan Mayor
  1. 1London

A combination of two drugs used to correct levels of the transmembrane protein that is defective or deficient in cystic fibrosis significantly improves patients’ lung function, show the results of two randomised studies published in the New England Journal of Medicine.1

The studies included a total of 1108 patients with cystic fibrosis who were homozygous for the commonest mutation in the gene coding for cystic fibrosis transmembrane conductance regulator (CFTR) protein, Phe508del. They were randomised to lumacaftor, which corrects cellular misprocessing …

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