A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial -

A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial
Information type: Randomised controlled trials
Source: The Lancet Respiratory Medicine
Specialities: Respiratory disorders
Summary
RCT (n=188) reported that combination of lumacaftor and ivacaftor improves FEV1 for patients with CF who are homozygous for phe508del CFTR, with a modest effect on sweat chloride concentration, thus supporting further exploration of this combination in this treatment setting.
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