Latest CFTR Stories

Data about defective regulatory protein described in Proceedings of the National Academy of Sciences A little more than a year after the FDA approved Kalydeco (Vx-770), the first drug of its kind to treat the underlying cause of cystic fibrosis, University of Missouri researchers believe they have found exactly how this drug works and how to improve its effectiveness in the future. Described in the current issue of the Proceedings of the National Academy of Sciences, MU researchers have...

TORONTO, June 26, 2012 /CNW/ - Rogers Media announced today the appointment of John Hinnen to Vice-President of News for Rogers Media Television and Radio, effective immediately.  In this newly created position, Hinnen assumes expanded duties to oversee the direction for all Rogers Media television and radio news brands nationally. Previously, Hinnen was Vice-President of News for Rogers Radio and Vice-President & General Manager of 680News, Canada's #1 news radio...

SOUTH PLAINFIELD, N.J., June 8, 2012 /PRNewswire/ -- PTC Therapeutics, Inc. today announced the results from a Phase 3 study of ataluren, an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). The results demonstrated positive trends in lung function as measured by FEV1 (forced expiratory volume in one second) and in the secondary outcome measure, rate of pulmonary exacerbations. Results also showed a substantial ataluren treatment effect in FEV1 and...

Researchers at Johns Hopkins have identified a gene that modifies the risk of newborns with cystic fibrosis (CF) developing neonatal intestinal obstruction, a potentially lethal complication of CF. Their findings, which appeared online March 15 in PLoS Genetics, along with the findings of their Toronto-based colleagues, published April 1 in Nature Genetics, may lead to a better understanding of how the intestines work and pave the way for identifying genes involved in secondary complications...

By developing software that uses 3-D models of proteins involved in cystic fibrosis, a team of scientists at Duke University has identified several new molecules that may ease the symptoms of the disease. Computer algorithms created by the team predict how well a given molecular structure will block a basic protein-protein interaction known to occur in cystic fibrosis. To test the predictions, the scientists synthesized the molecules and measured how well they attached to one of the...

Cystic fibrosis (CF) is a devastating disease caused by mutations in the CFTR gene. In Canada, one in every 3,600 children born has the disease. Researchers have long been puzzled as to how individuals who carry the same CFTR mutations can experience such different courses of disease. Patients with CF are affected in multiple organs such as the lungs, pancreas and liver, to varying degrees. An international team led by The Hospital for Sick Children (SickKids) and the University for...

University of North Carolina at Chapel Hill researchers working as part of the International Cystic Fibrosis Consortium have discovered several regions of the genome that may predispose cystic fibrosis (CF) patients to develop an intestinal blockage while still in the uterus. A report of this international study appears online April 1, 2012 in the journal Nature Genetics. It was the work of the North America CF Gene Modifier Consortium, which brought together dozens of investigators from...

New research could provide roadmap for more effective drug discovery for CF A recent study led by Gergely Lukacs, a professor at McGill University's Faculty of Medicine, Department of Physiology, and published in the January issue of Cell, has shown that restoring normal function to the mutant gene product responsible for cystic fibrosis (CF) requires correcting two distinct structural defects. This finding could point to more effective therapeutic strategies for CF in the future. CF, a...

Breakthrough therapy targets defective protein SILVER SPRING, Md., Jan. 31, 2012 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration today approved Kalydeco (ivacaftor) for the treatment of a rare form of cystic fibrosis (CF) in patients ages 6 years and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. (Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO) CF is a serious genetic disorder affecting the lungs...

Final stage clinical trial shows significantly improved lung function in subset of CF patients A new study has confirmed that the drug, ivacaftor (VX-770), significantly improves lung function in some people with cystic fibrosis (CF). The results of the phase III clinical trial study, "A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation," led by Bonnie W. Ramsey, MD of Seattle Children's Research Institute and the University of Washington, were published today in the...