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Clinical effect of lumacaftor/ivacaftor in F508del homozygous CF patients with FEV(1) 90% predicted at baseline.
Aalbers BL, de Winter-de Groot KM, Arets HGM, Hofland RW, de Kiviet AC, van Oirschot-van de Ven MMM, Kruijswijk MA, Schotman S, Michel S, van der Ent CK, Heijerman HGM. Aalbers BL, et al. J Cyst Fibros. 2020 Jul;19(4):654-658. doi: 10.1016/j.jcf.2019.12.015. Epub 2020 Jan 7. J Cyst Fibros. 2020. PMID: 31924546
OBJECTIVE: The first available CFTR modulator combination for homozygous F508del patients, lumacaftor/ivacaftor, has not been tested in patients with percentage predicted (pp)FEV(1) > 90 in the phase III trials. ...Treatm …
OBJECTIVE: The first available CFTR modulator combination for homozygous F508del patients, lumacaftor/ivacaft
Lumacaftor/ivacaftor, a novel agent for the treatment of cystic fibrosis patients who are homozygous for the F580del CFTR mutation.
Bulloch MN, Hanna C, Giovane R. Bulloch MN, et al. Expert Rev Clin Pharmacol. 2017 Oct;10(10):1055-1072. doi: 10.1080/17512433.2017.1378094. Epub 2017 Sep 22. Expert Rev Clin Pharmacol. 2017. PMID: 28891346 Review.
Approximately 73% of patients are homozygous for the F508del cystic fibrosis transmembrane conductance regulator [CFTR] mutation. ...Areas covered: In 2016, the Food and Drug Administration and the European Commission approved LUM/IVA (Orkambi), a CFTR modula …
Approximately 73% of patients are homozygous for the F508del cystic fibrosis transmembrane conductance regulator [CFTR] …