Addressing variability in newborn screening can prevent missed diagnoses and delays in early care intervention.
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CF Foundation President and CEO Robert J. Beall, Ph.D. testified today before the House of Representatives' Energy and Commerce Committee at a hearing entitled, “21st Century Cures: Incorporating the Patient Perspective.”
Cystic fibrosis is a genetic disorder that affects the lungs, pancreas, and other organs. Keep reading to learn how to treat and live with CF.
The Cystic Fibrosis Foundation awarded Talee Bio Inc. up to $4.5 million to develop methods for delivering potential gene therapy treatments to the lungs.
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.
The Cystic Fibrosis Foundation awarded up to $5.1 million to TB Alliance to advance a drug discovery program in partnership with Johns Hopkins University. The effort will identify compounds with the potential to treat nontuberculous mycobacteria infections that are increasingly found among people with CF.
The U.S. Food and Drug Administration has approved the use of tezacaftor/ivacaftor (Symdeko®) for children with cystic fibrosis ages 6 to 11 with specific mutations.
Vertex Pharmaceuticals Inc. today announced that it will pursue FDA approval for the triple-combination therapy VX-445 (elexacaftor) plus tezacaftor/ivacaftor (Symdeko®).
Today, Vertex released positive preliminary clinical trial results for the potential triple-combination therapy VX-445 plus tezacaftor/ivacaftor (Symdeko®).
The Institute for Clinical and Economic Review (ICER) recently posted a draft scoping document to outline their planned assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The document is open for public comment through October 21.