The U.S. Food and Drug Administration approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 2 to 5 who have two copies of the F508del mutation.
Researchers are exploring treatments that will keep people with cystic fibrosis as healthy as possible until a cure is found. In the first plenary at the NACFC, two CF scientists explain the progress of current research.
For those of us without a medication that targets our cystic fibrosis mutation, it's important to remember that our life with CF is a marathon.
The CF Foundation is focused on bringing forward new therapies to help people with CF enjoy the best health and quality of life.
New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies.