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With this approval, approximately 2,250 children in the U.S. will be eligible for Trikafta® (elexacaftor/tezacaftor/ivacaftor), including more than 900 who will have access to a CFTR modulator for the first time. The Cystic Fibrosis Foundation believes it is beneficial to start people with cystic fibrosis on modulators as early as possible to help prevent lung damage and the onset of complications.
Proposed policies would impose significant barriers and add burden for people with CF
Groups urging need for revitalizing antimicrobial development call for passage of the bill this year
The U.S. Food and Drug Administration approved Kalydeco® (ivacaftor) for infants as young as 1 month who have at least one copy of 97 mutations that have proven to be responsive to Kalydeco.
Medicaid beneficiaries are encouraged to update their information with Medicaid to ensure continued coverage.
The Cystic Fibrosis Foundation is providing additional funds to TB Alliance to advance the development of a compound that could be used to treat infections caused by nontuberculous mycobacteria.
The Cystic Fibrosis Foundation is providing additional funding to Arcturus Therapeutics to develop an inhaled messenger RNA therapy for CF that provides lung cells with the correct instructions to make functional cystic fibrosis transmembrane conductance regulator protein. The funding brings our total commitment to Arcturus to approximately $25 million.
Centering the patient experience is the only way to successfully reform pharmacy benefit manager practices
More than 60 teens from across the country — almost half of whom live with cystic fibrosis — advocated for support of the PASTEUR Act.