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 Caucasian populations. CF is a progressive disease that affects many organ systems, but most of its morbidity and mortality are associated with its impact on the respiratory system.
Interventions of Interest:
- Tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals)
- Lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals)
- Ivacaftor (Kalydeco®, Vertex Pharmaceuticals)
University of Missouri-St. Louis
Century BC Room, Millennium Student Center
1 University Boulevard
St. Louis, MO 63121
The Midwest CEPAC will convene to discuss treatments for cystic fibrosis.
Open to public comment from March 15 – April 12, 2018. For more information on submitting a public comment, please visit our stakeholder engagement page.