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.
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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.
Carrier (or genetic) testing not only plays a key role in the diagnosis of cystic fibrosis, but testing also allows parents to find out what their chances are of having a child with CF to help inform important family planning decisions.
The Cystic Fibrosis Foundation recently hosted the CFF Research Conference: Pushing the Frontiers, bringing together nearly 150 basic scientists, clinical researchers, graduate students and pharmaceutical company representatives to share information on recent advances and critical challenges in CF drug discovery and development.
The Cystic Fibrosis Foundation has awarded more than $23 million across 11 sites in its Research Development Program (RDP), a network of research centers that brings together top-notch scientists from different disciplines to apply their expertise to the challenges of treating cystic fibrosis.