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Metabolomic responses to lumacaftor/ivacaftor in cystic fibrosis
Pediatric Pulmonology, 2018AbstractBackgroundCystic fibrosis (CF) is a life‐limiting disease caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Lumacaftor/Ivacaftor is a novel CFTR modulator approved for patients that are homozygous for Phe508del CFTR, but its clinical effectiveness varies amongst patients, making it difficult to determine
Benjamin T. Kopp +6 more
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Lumacaftor/Ivacaftor: A Review in Cystic Fibrosis
Drugs, 2016Lumacaftor/ivacaftor (Orkambi™) is a fixed-dose tablet containing a corrector (lumacaftor) and potentiator (ivacaftor) of the cystic fibrosis transmembrane conductance regulator (CFTR) and is the first therapy approved to treat the underlying cause of cystic fibrosis in patients (aged ≥12 years) homozygous for the most common CFTR mutation, F508del ...
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Real-Life Safety and Effectiveness of Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis
American Journal of Respiratory and Critical Care Medicine, 2020Marlene Murris-Espin +2 more
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