Steve Bryson, PhD, science writer —

Two non-invasive tests — MRI scans and the lung clearance index (LCI) — were more sensitive to early lung disease in children with cystic fibrosis (CF) than standard lung function tests, a study from Switzerland reports. Better ways of evaluating lung health in infants and children are needed,…

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,…

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…

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…

Despite some similarities, the lung composition of microbes and their antimicrobial resistance profiles differed between people with bronchiectasis and cystic fibrosis (CF) and those with non-CF bronchiectasis, a study found. The findings suggest “the need for customized management strategies for each disease,” the researchers wrote in the study, “…

Scientists at the Broad Institute of MIT and Harvard have created a gene editing technology that may be able to insert or substitute entire genes in human cells, which could pave the way for new gene therapies for diseases such as cystic fibrosis (CF). The approach could efficiently insert…

CF AMR Syndicate, a U.K. research support group, has awarded BioVersys up to £500,000 ($636,000) to develop small molecules targeting hard-to-treat nontuberculous mycobacteria (NTM) infections in people with cystic fibrosis (CF). Established in 2019 as a cross-sector (e.g., public-private) initiative with the Cystic Fibrosis Trust to…

A single dose of 4D Molecular Therapeutics’ inhaled gene therapy candidate 4D-710 improved or stabilized lung function in adults with cystic fibrosis (CF), according to interim data from a Phase 1/2 clinical trial. Additional data from the AEROW (NCT05248230) study, which is evaluating the therapy in CF…