The Carolyn and C. Richard Mattingly Leadership in Mental Health Care Award honors those who are committed to the mental health of people with cystic fibrosis.
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Cystic Fibrosis Foundation Therapeutics Inc. announced an award of up to $5M to Editas Medicine Inc. to advance potential gene editing approaches for cystic fibrosis.
Researchers' catalog of airway cell types could reveal targets for future genetic therapies
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.
Cystic fibrosis biological samples are available to qualified researchers to help develop promising new studies that will support CF research and aid in drug development and drug discovery.
The goal of the CFTR Chemical Compound program is to enable cystic fibrosis research scientists to test known cystic fibrosis transmembrane conductance regulator (CFTR) modulating compounds in different functional assays.
Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.
Dr. Frank Accurso, director of cystic fibrosis clinical research at Children's Hospital Colorado and University of Colorado Denver, participated in a congressional roundtable discussion at National Jewish Health in Denver, Colorado.
Every person has two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. A person must inherit two copies of the CFTR gene that contain mutations — one copy from each parent — to have cystic fibrosis.