Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation
G Spencer-Green, Michael W. Konstan, Steven M. Rowe, Manfred Ballmann, Laurent Vernillet, Jordan Dunitz, Jeffrey Clancy, Preston W. Campbell, Inez Bronsveld, Michael S. Schechter, Moira L. Aitken, Steve Wisseh, Melissa A. Ashlock, Frank J. Accurso, Richard B. Moss, Joseph M. Pilewski, Manu Jain, Ronald C. Rubenstein, K. De Boeck, Martyn C. Botfield, Anissa Leonard, Henry L. Dorkin, Michael Boyle, Scott H. Donaldson, Karen McCoy, Daniel Rosenbluth, Peter R. Durie, Claudia L Ordoñez, Karl Yen, Raouf Amin
Abstract
VX-809, a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, has been shown to increase the cell surface density of functional F508del-CFTR in vitro.
Topics & Concepts
Cystic fibrosisMedicineMutationCompound heterozygosityPharmacologyInternal medicineGeneticsBiologyGeneCystic Fibrosis Research Advances