News

Mutations in the CFTR gene that associate with severe disease can also lead to cardiac problems — specifically, the development of subclinical left ventricular dysfunction — in cystic fibrosis patients, a study suggests. The study, “Subclinical Left Ventricular Dysfunction is Influenced by Genotype Severity in Patients with Cystic…

Vertex Pharmaceuticals has completed enrollment for two Phase 3 studies testing the triple combination therapy of its new CFTR corrector VX-659 together with tezacaftor (VX-661) and Kalydeco (ivacaftor) in patients with cystic fibrosis (CF). The studies focus on patients with two F508del mutations in the CFTR gene (the…

Editor’s Note: This is the second of a four-part series looking at Vertex Pharmaceuticals and its “all in for CF” approach. Here, we detail the steps that made three disease-modifying oral treatments for cystic fibrosis possible. Kalydeco (ivacaftor), as Vertex‘s cystic fibrosis potentiator came to be known, was a small…

Beginning Oct. 1, the cystic fibrosis (CF) targeted therapy Orkambi (lumacaftor/ivacaftor) will be included on Australia’s Pharmaceutical Benefits Scheme. The Australian government’s decision resulted from the completion of an agreement with Orkambi’s manufacturer, Vertex Pharmaceuticals, and was supported by the recent positive recommendation from the Pharmaceutical Benefits Advisory Committee…

A research team at The University of Queensland was honored for its work on the spread of bacteria among cystic fibrosis patients, being named a winner of this year’s Australian Museum Eureka Prizes. The prizes recognize excellence in four fields:  research and innovation, science engagement, science leadership, and science education. CF Air, as…

Vertex Pharmaceuticals and Genomics announced a three-year collaboration to better understand genetic variations and their effect on clinical outcomes of diseases with unmet therapeutic needs, including cystic fibrosis. The collaboration also will promote the development of precision medicine, an approach to customize treatment based on each patient’s genetic…

Using a computer modeling method, a research team at the University of Copenhagen, Denmark, demonstrated some of the early developmental mechanisms involved in the formation of the pancreas’ network of ducts. The findings of the study, conducted in mice, offer new knowledge of how the pancreas works, and could…

Up to one month of treatment with inhaled vancomycin, the antibiotic of choice for treating most methicillin-resistant Staphylococcus aureus (MRSA) infections, together with a combination of oral and topical antibiotics, failed to clear infection in cystic fibrosis (CF) patients at better rates than the antibiotic combination alone, results from a Phase 2 clinical…

Blood levels of D-lactate may be markers of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF), according to preliminary data from a new study. The research, “Serum Level of D-Lactate in Patients with Cystic Fibrosis: Preliminary Data,” was published in the journal…

Single nucleotide polimorphisms (or variants), known as SNPs — the most common type of genetic variation — in the SLC26A9 gene are linked to poorer lung responses to Kalydeco in cystic fibrosis (CF) patients, a French study reports. The study, “SLC26A9 Gene Is Associated With Lung Function Response to Ivacaftor in Patients With…