cystic fibrosis

Cystic Fibrosis Foundation leaders and cystic fibrosis (CF) community advocates met at the White House with President Barack Obama, officials from the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA), and other CF stakeholders last month for the Precision Medicine Initiative (PMI) Summit. The summit was an opportunity…

Collaborative research between Eindhoven University of Technology (Netherlands), McGill University (Canada), and the University of Duisburg-Essen (Germany) has identified a promising approach that could lead to the development of an efficient treatment for cystic fibrosis (CF). The paper, “Characterization and small-molecule stabilization of the multisite tandem binding between 14-3-3…

Researchers identified a novel, rare mutation causing cystic fibrosis (CF) in a pediatric African-American patient. The mutation, however, was found to be responsive to the CFTR corrector VX-809, therefore identifying potential personalized therapeutics. The study, “c.3623G > A mutation encodes a CFTR protein with impaired channel function,” was published…

Arch Biopartners, Inc, announced that it recently submitted an orphan drug designation application to the European Medicines Authority (EMA) for its product AB569, a potential treatment of pulmonary infections caused by Pseudomonas aeruginosa in patients with cystic fibrosis (CF). The EMA is now reviewing the application, and the Committee for Orphan Medicinal Products…

A new study published in the journal e-LIFE has taken a novel approach to looking at deficits in protein folding associated with mutations in the CF transmembrane conductance regulator (CFTR) gene, the underlying cause of cystic fibrosis (CF). By identifying the signaling networks controlling protein folding, the study opens…

Cystic Fibrosis News Today has covered news of the latest treatments, events, clinical trials, and research updates in cystic fibrosis on a daily basis throughout the past year. As 2015 comes to an end, here are 10 articles most widely read by Cystic Fibrosis News Today readers, each with a brief summary of the developments that…

A new study of children with cystic fibrosis found that co-infection with methicillin-resistant bacteria may be associated with increased pulmonary morbidity, especially in children with repeat hospitalizations. The retrospective study, conducted at a U.S. pediatric medical center with high rates of methicillin-resistant respiratory infections, is titled “Clinical characteristics and…

In a new study, researchers investigated the CFTR gene locus (its location and position in DNA) and the role of elements involved in regulating the chromatin structure and gene expression, discovering a mechanism that coordinates these regulatory elements and maintains normal gene expression levels when structural perturbations occur. The findings…

A Case Western Reserve University researcher is starting two studies, based on angiotensin signaling and gene “correction,” to advance research into more effective ways of treating cystic fibrosis (CF) and to better understand the ideal timing for such interventions. Both studies will begin in January. CF is due to a defective gene that causes a thick buildup…

A new study in The Journal of Molecular Diagnostics found that the genetic screening panel for cystic fibrosis (CF) — based on a list of 23 known mutations — is not adequate for diagnosing nonwhite populations. CF is caused by mutations in the transmembrane conductance regulator (CFTR)…