Article
Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR.
Annals of the American Thoracic Society - 1 Feb 2017
Rowe Steven M, McColley Susanna A, Rietschel Ernst, Li Xiaolei, Bell Scott C, Konstan Michael W, Marigowda Gautham, Waltz David, Boyle Michael P
Abstract excerpt
RATIONALE: In a prior study, lumacaftor/ivacaftor treatment (≤28 d) in patients with cystic fibrosis (CF) heterozygous for F508del-CFTR did not improve lung function. OBJECTIVES: To evaluate an optimized lumacaftor/ivacaftor dosing regimen with a longer duration in a cohort of patients heterozygous for F508del-CFTR. METHODS: Patients aged 18 years or older with a confirmed CF diagnosis and percent predicted FEV1...
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