News

Lung transplant rates among people with cystic fibrosis (CF) in the U.S. have declined dramatically since the approval of Trikafta (elexacaftor/tezacaftor/ivacaftor), a study found. However, almost half of patients who received transplants in recent years were on Trikafta before their procedures. Moreover, a notable proportion of CF patients…

Nearly two-thirds of cystic fibrosis (CF) patients in a study showed signs of type 2 inflammation, which was associated with worse lung function, more infections, and a higher risk of death. This form of inflammation, most commonly linked to allergic conditions, was reduced somewhat after treatment with CFTR…

Sionna Therapeutics has gained the rights to develop and market three of AbbVie’s clinical-stage compounds, a move that expands the company’s pipeline of small molecules that are designed to restore the function of the CFTR protein, which is faulty or absent in cystic fibrosis (CF). The compounds…

A woman and her daughter were diagnosed with chronic rhinosinusitis, an inflammatory condition of the sinuses and nasal passages, with features matching cystic fibrosis (CF), a case study reports. However, neither patient carried mutations in the CFTR gene that cause CF. The pair also presented with signs and…

One year of Trikafta treatment improved lung function, reduced abnormalities in lung structure, and halted disease progression among school-age children, ages 6-11, with cystic fibrosis (CF), according to a real-world analysis. “The majority of the children even achieved normal lung function with the triple combination therapy. That’s a…

Using a newly enhanced gene-editing technology called prime editing, researchers in the U.S. have efficiently corrected the most common mutation that causes cystic fibrosis (CF) in human lung cells. By correcting this mutation, known as F508del, in the CFTR gene, scientists at the Broad Institute of MIT and Harvard,…

In a real-world study involving children with cystic fibrosis (CF) in Italy, Kaftrio — an approved CF therapy sold as Trikafta in North America — was shown to lead to significant improvements in lung function for more…

Despite treatment with Kalydeco (ivacaftor), females with cystic fibrosis (CF) continue to experience more lung exacerbations — periods when CF respiratory symptoms suddenly worsen — than do males, for reasons that are not well understood, a large patient registry study reported. “Our findings demonstrate that sex disparities…

Despite recent guidelines recommending nutrient consumption similar to the general population, many adults and late adolescents with cystic fibrosis (CF) still follow a high-fat, high-energy diet, according to a review done by researchers in Australia. The findings suggest that “overall, diet intakes of adults with CF appear to be…

Arcturus Therapeutics plans to soon submit an application to the U.S. Food and Drug Administration (FDA) seeking permission to begin testing multiple ascending doses of ARCT-032, an inhaled experimental treatment for cystic fibrosis (CF). The planned Phase 2 study, supported by promising safety and preliminary efficacy data…