Article
An Observational Study of Outcomes and Tolerances in Patients with Cystic Fibrosis Initiated on Lumacaftor/Ivacaftor.
Annals of the American Thoracic Society - 1 Nov 2017
Jennings Mark T, Dezube Rebecca, Paranjape Shruti, West Natalie E, Hong Gina, Braun Andrew, Grant Jonathan, Merlo Christian A, Lechtzin Noah
Abstract excerpt
RATIONALE: In July 2015, the U.S. Food and Drug Administration approved lumacaftor/ivacaftor for use in patients with cystic fibrosis (CF). This drug targets the primary defect in the CFTR protein that is conferred by the F508del CFTR mutation. OBJECTIVE: As there is limited experience with this therapy outside of clinical trials, this study aims to examine the clinical experience of this new drug in a population...
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