Cystic Fibrosis

Midwest CEPAC

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)

Associated Meetings

May 17, 2018 9:30am-3:30pm CT

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.


Key Dates

Associated Materials

10/11/2017 – 10/27/2017

10/31/2017

10/31/2017

10/31/2017 – 11/20/2017

11/30/2017

02/07/2018

02/07/2018

03/15/2018

Open to public comment from March 15 – April 12, 2018. For more information on submitting a public comment, please visit our stakeholder engagement page.