Answer
Ivacaftor potentiates CFTR in vitro by increasing channel gating (open probability), enhancing chloride transport in specified gating mutations, and also potentiates R117H-CFTR; the exact mechanism is not fully understood, and in vitro responses do not necessarily predict in vivo effects or clinical benefit.
Sources for this answer
- Kalydeco 150 mg film-coated tabletsVertex Pharmaceuticals (Europe) Limited · emc · SmPC §5.1 Pharmacodynamic properties · Version 35Document updated
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