A record 5,000 people from dozens of countries will gather next month in Denver for the 32nd Annual North American Cystic Fibrosis Conference (NACFC) — the world’s largest event focused on cystic fibrosis (CF) research and care. The Oct. 18-20 event at Denver’s Colorado Convention Center features 24 symposium sessions, 31…
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Dietary Flavonoids May Help in Managing CF Through Impact on Gut Bacteria, Small Study Suggests
Consuming dietary flavonoids — compounds naturally found in fruits, vegetables, chocolate, and beverages like wine and tea — may be associated with changes in gut bacteria that potentially affect the body’s metabolism, immune function, and inflammation in patients with cystic fibrosis (CF), a small…
FDA Gives Green Light to 2 Trials for AB-PA01, Potential Therapy Against P. aeruginosa Infections
The U.S. Food and Drug Administration gave positive feedback to the design of AmpliPhi Biosciences’ two proposed clinical trials of its Pseudomonas aeruginosa-targeting treatment candidate AB-PA01, the company announced in a press release. P. aeruginosa is the major cause of lung infections in cystic fibrosis patients.
A new electronic nose, called Aeonose, was seen to be a feasible, accurate and easy-to-use diagnostic test for cystic fibrosis (CF) in children and adolescents, a small pilot study shows. The research “Feasibility and diagnostic accuracy of an…
Blocking superoxide dismutase, the enzyme that responds to stress, in Pseudomonas aeruginosa bacteria improves antibiotics’ efficacy, a Canadian study reported. The study, “Superoxide dismutase activity confers (p)ppGpp-mediated antibiotic tolerance to stationary-phase Pseudomonas aeruginosa,” was published in Proceedings of the National Academy of Sciences (PNAS). “We identified a new function important…
Orkambi Eases Flares Even in CF Patients Slow to Show Improvement in Lung Health, Trial Data Show
Cystic fibrosis patients who failed to show early improvements in lung function while being treated with Orkambi (lumacaftor/ivacaftor) still experienced fewer pulmonary exacerbations than those given a placebo in Phase 3 studies, a pooled analysis of those trials reports. Clinicians should consider the totality of outcomes when examining the…
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…
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