Here's what you need to know about the four CFTR (cystic fibrosis transmembrane conductance regulator) modulators.
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.
As the mother of a child with CF and volunteer for the CF Foundation, I know first-hand the extraordinary things that Dr. Bob Beall has done for our community, and I am grateful for the legacy of hope that he leaves behind.
Earlier this month, we learned that the new CF drug Orkambi was approved by the FDA. This was such an exciting, memorable and emotional day for everyone in the CF community. We heard from many who were thrilled about the potential for this drug to help people with CF and also heard from those who would not benefit from this breakthrough. Following are some reactions from those this impacts the most: people with CF and their loved ones.
The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
I've lived my whole life like it has an early expiration date. Recently, a drug was FDA-approved that could change that. I'm grateful, but I'm also scared…
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.