News

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…

Authorities in the U.S. and Europe are reviewing applications from Vertex Pharmaceuticals seeking approval of the company’s novel CFTR modulator triple combination, vanzacaftor, tezacaftor and deutivacaftor.  Vertex is asking that the vanza combo be approved for people with cystic fibrosis (CF) ages 6 and older who carry…

Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly lowered several pro-inflammatory blood markers with as early as one month of use in cystic fibrosis (CF) patients carrying F508del — the most common CF-causing mutation — and in parallel with gains in lung function. These are the findings of a small study that suggest…

Tezacaftor, a medication used to treat cystic fibrosis (CF), may unexpectedly trap certain fats in the lungs, diverting their use as chemical raw material that the body converts into ceramides, which are needed for the brain to develop and respond to stress, according to…

Among young children with cystic fibrosis (CF), starting the approved oral therapy Orkambi (ivacaftor/lumacaftor) led to favorable changes in nutrition and growth status during the first six months of treatment, according to a small real-world study. Orkambi, first approved in the U.S. in 2015 and now indicated for…

Danish children with cystic fibrosis (CF) born between 2000 and 2022 showed noticeable gains in growth during their first five years of life, with those born after 2016 seeing the most progress in reaching normal growth standards, a study shows. This is likely due to newborn screening programs (NBS)…