Cystic Fibrosis
Interventions of Interest: Tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals) Lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals) Ivacaftor (Kalydeco®, Vertex Pharmaceuticals) Elexacaftor/tezacaftor/ivacaftor (Trikafta®, Vertex Pharmaceuticals) Cystic fibrosis is an autosomal recessive condition caused by mutations in the CFTR gene. It is relatively rare, occurring in approximately 1 in 2,500 to 3,000 livebirths, but is the most common, lethal genetic disease in […]