1. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508delmutation: a double-blind, randomised, phase 3 trial
- Author
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Heijerman, Harry G M, McKone, Edward F, Downey, Damian G, Van Braeckel, Eva, Rowe, Steven M, Tullis, Elizabeth, Mall, Marcus A, Welter, John J, Ramsey, Bonnie W, McKee, Charlotte M, Marigowda, Gautham, Moskowitz, Samuel M, Waltz, David, Sosnay, Patrick R, Simard, Christopher, Ahluwalia, Neil, Xuan, Fengjuan, Zhang, Yaohua, Taylor-Cousar, Jennifer L, McCoy, Karen S, McCoy, Karen, Donaldson, Scott, Walker, Seth, Chmiel, James, Rubenstein, Ronald, Froh, Deborah K., Neuringer, Isabel, Jain, Manu, Moffett, Kathryn, Taylor-Cousar, Jennifer L., Barnett, Bruce, Mueller, Gary, Flume, Patrick, Livingston, Floyd, Mehdi, Nighat, Teneback, Charlotte, Welter, John, Jain, Raksha, Kissner, Dana, Patel, Kapilkumar, Calimano, Francisco J., Johannes, Jimmy, Daines, Cori, Keens, Thomas, Scher, Herschel, Chittivelu, Subramanyam, Reddivalam, Sudhakar, Klingsberg, Ross Carl, Johnson, Larry G., Verhulst, Stijn, Macedo, Patricia, Downey, Damien, Connett, Gary, Nash, Edward, Withers, Nicholas, Lee, Timothy, Bakker, Marleen, Heijerman, Harry, Vermeulen, Francois, Van Braeckel, Eva, Knoop, Christiane, De Wachter, Elke, van der Meer, Renske, Merkus, Petrus, and Majoor, Christof
- Abstract
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have been achieved with the combination of a CFTR corrector and potentiator in people with cystic fibrosis homozygous for the F508delmutation. The addition of elexacaftor (VX-445), a next-generation CFTR corrector, to tezacaftor plus ivacaftor further improved F508del-CFTR function and clinical outcomes in a phase 2 study in people with cystic fibrosis homozygous for the F508delmutation.
- Published
- 2019
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