Researchers' catalog of airway cell types could reveal targets for future genetic therapies
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The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children ages 6 through 11 who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.
Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021.
Foundation welcomes new members to advance its mission
The Cystic Fibrosis Foundation recently submitted comments to the National Institutes of Health on improving racial equity, diversity, and inclusion in the biomedical research workforce.
FDA issues emergency use authorization for a third COVID-19 vaccine.
The Cystic Fibrosis Foundation has renewed its commitment to supporting the health and well-being of adults living with cystic fibrosis by supporting free access to the online exercise, education, and well-being platform, Beam.