Phase 3 Results from Two Studies of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Triple Combination Treatment for Cystic Fibrosis Concurrently Published in The New England Journal of Medicine and The Lancet - Seite 3

An open-label extension study of tezacaftor/ivacaftor (TEZ/IVA) therapy in patients aged >12 years with cystic fibrosis (CF) homozygous for F508del-CFTR (F/F) or heterozygous for F508del-CFTR and a residual function mutation (F/RF)

Late-Breaking Poster #853

Patrick Flume

Thursday, October 31 11:15 a.m. to 1:45 p.m.

CF Disease Burden

Development of the cystic fibrosis questionnaire-revised preference based scoring algorithm (CFQ-R-8D)

Poster #758

Lisa McGarry

Thursday, October 31 11:15 a.m. to 1:45 p.m.

Burden of illness in patients >12 years with cystic fibrosis heterozygous for the F508del mutation and a minimal function mutation

Poster #220

Greg Sawicki

Thursday, October 31 11:15 a.m. to 1:45 p.m.

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.