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.
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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.
In an international research project, scientists are examining cystic fibrosis transmembrane conductance regulator (CFTR) mutations to determine which ones cause CF and to provide additional information associated with these mutations. Their findings are available in an online searchable database.
The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.
The Cystic Fibrosis Foundation invited more than 100 scientists to its research conference in Savannah, Ga., last month to discuss recent advancements in gene therapy, gene editing and stem-cell biology and how these new technologies could be applied to finding a one-time cure for cystic fibrosis.
The Associated Press published a story this week highlighting the Cystic Fibrosis Foundation's "groundbreaking" drug development model and its role in developing CF treatments that target the root cause of the disease.
The Cystic Fibrosis Foundation has entered into an agreement with Synspira Therapeutics Inc. to develop a non-porcine enzyme replacement therapy to offer an alternative to people with cystic fibrosis who cannot digest food properly.
At the 2016 North American Cystic Fibrosis Conference (NACFC) held on Oct. 27-29 in Orlando, Fla., the Cystic Fibrosis Foundation announced the recipients of seven awards given to members of the research and medical community whose achievements have helped advance cystic fibrosis research and care.