This year’s awardees showcase the diversity of journeys in the cystic fibrosis community.
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Orkambi® is now approved for use in children with cystic fibrosis ages 1-2 years who have two F508del mutations. This is the only modulator available to very young children with these mutations.
The U.S. Food and Drug Administration (FDA) approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 6 to 11, who have two copies of the F508del mutation.
The U.S. Food and Drug Administration (FDA) has decided not to approve the use of ivacaftor (Kalydeco®) to treat people with cystic fibrosis ages 2 and older who have one of 23 rare CF mutations, known as “residual function” mutations.
The Cystic Fibrosis Foundation celebrates the FDA's approval today of the lumacaftor/ivacaftor combination drug (Orkambi™) as an important advance for the cystic fibrosis community. Orkambi targets the underlying cause of the disease in people with two copies of the most common CF gene mutation.
The U.S. Food and Drug Administration has approved the use of the triple-combination modulator elexacaftor/tezacaftor/ivacaftor (Trikafta™) for people with cystic fibrosis ages 12 and older who have at least one copy of the F508del mutation.
Recipients include five innovative programs from around the country that empower the CF community.
Ray Poole and Julie Riedy to lead efforts in Washington, D.C., for volunteer conference, Until CF Stands for Cure Found.
The Cystic Fibrosis Foundation presented five outstanding members of the CF community with awards at the 2019 Volunteer Leadership Conference. These included the Tomorrow's Leader Award, the Jena Award, the Alex Award, and the Dream Big Award.