CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials

Scott C. Bell, Peter J. Barry, Kris De Boeck, Pavel Drevinek, J. Stuart Elborn, Barry J. Plant, Predag Minić, Eva Van Braeckel, Stijn Verhulst, Karine Muller, Desirée Kanters, Susan Bellaire (+5 others)
2019 Journal of Cystic Fibrosis  
Several treatment approaches in cystic fibrosis (CF) aim to correct CF transmembrane conductance regulator (CFTR) function; the efficacy of each approach is dependent on the mutation(s) present. A need remains for more effective treatments to correct functional deficits caused by the F508del mutation.
doi:10.1016/j.jcf.2019.04.014 pmid:31056441 fatcat:6nar6hl5grhzdbu3uayunrq5ma