Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.

Peter Gordon MiddletonMarcus A MallPavel DřevínekLarry C LandsEdward F McKoneDeepika PolineniBonnie W RamseyJennifer L Taylor-CousarElizabeth TullisFrançois VermeulenGautham MarigowdaCharlotte M McKeeSamuel M MoskowitzNitin NairJessica SavageChristopher SimardSimon TianDavid WaltzFengjuan XuanSteven M RoweRaksha Jainnull null

Published in: The New England journal of medicine (2019)

Elexacaftor-tezacaftor-ivacaftor was efficacious in patients with cystic fibrosis with Phe508del-minimal function genotypes, in whom previous CFTR modulator regimens were ineffective. (Funded by Vertex Pharmaceuticals; VX17-445-102 ClinicalTrials.gov number, NCT03525444.).

Keyphrases

cystic fibrosis
pseudomonas aeruginosa
lung function
end stage renal disease
ejection fraction
newly diagnosed
peritoneal dialysis
patient reported outcomes