- Article
Rescue of Mutant CFTR Channel Activity by Investigational Co-Potentiator Therapy
- Mafalda Bacalhau,
- Filipa C. Ferreira,
- Marcelo Folhadella M. F. Azevedo,
- Talita P. Rosa,
- Camilla D. Buarque and
- Miquéias Lopes-Pacheco
Background: The potentiator VX-770 (ivacaftor) has been approved as a monotherapy for over 95 cystic fibrosis (CF)-causing variants associated with gating/conductance defects of the CF transmembrane conductance regulator (CFTR) channel. However, desp...