Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function

Cystic fibrosis (CF) is a genetic autosomal recessive disease involving mutations in the gene encoding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein [1-3]. CFTR dysfunction is responsible for a multisystem disease dominated by respiratory manifestations with chronic airway infection, accelerated decline in lung function and frequent respiratory exacerbations, and by impaired nutritional status [1, 2]. Over the past decades, CF management has consisted in symptomatic treatment, which includes airway clearance techniques, systemic and inhaled antibiotics, pancreatic enzyme replacement and high fat-high calorie diet [2, 4].

Source: Journal of Cystic Fibrosis - June 22, 2020 Category: Respiratory Medicine Authors: Pierre-R égis Burgel, Isabelle Durieu, Raphaël Chiron, Laurent Mely, Anne Prevotat, Marlene Murris-Espin, Michele Porzio, Michel Abely, Philippe Reix, Christophe Marguet, Julie Macey, Isabelle Sermet-Gaudelus, Harriet Corvol, Stéphanie Bui, Tiphaine Bi Tags: Original Article Source Type: research