Cystic Fibrosis is a complex disease caused by mutations in the CFTR gene. These mutations influence how patients experience the disease, so understanding the genetics is the key to understanding CF and its treatment.
Information on cystic fibrosis-causing mutations
Information about lumacaftor/ivacaftor phase III clinical trials
Downloadable ivacaftor resources
1. ORKAMBI Summary of Product Characteristics. January 2018. Vertex Pharmaceuticals (Europe) Limited.