News

Researchers have developed a device that could improve gene therapy delivery to the lungs for people with genetic conditions such as cystic fibrosis (CF). In a series of preclinical experiments, the research team showed that its device could deliver a therapeutic payload to cells better than a standard inhaler…

Most parents of newborns diagnosed with cystic fibrosis (CF) or a different disease as part of U.S. newborn screening programs said understanding the test results was difficult and about a third said they lacked support from their child’s healthcare team, according to a national survey.

Younger adults with cystic fibrosis (CF) and a history of frequent pulmonary exacerbations are more likely than other adult patients to have put on weight six months into treatment with Trikafta (ivacaftor/tezacaftor/elexacaftor), a study found. The study, “Predicting weight gain in patients with cystic fibrosis on triple…

Treatment with the antibiotic azithromycin reduces abnormal thickening of the walls of bronchi — air passages leading to the lungs — in young children with cystic fibrosis (CF), and this may translate into smoother blood flow to the lungs, a study suggests. Data come from COMBAT-CF (NCT01270074),…

Infections of Pseudomonas aeruginosa usually persist in people with cystic fibrosis (CF) after Trikafta is started, a study reports. The study, “Persistence and evolution of Pseudomonas aeruginosa following initiation of highly effective modulator therapy in cystic fibrosis,” was published in mBio. CF is…

This year, Cystic Fibrosis Awareness Month events are celebrating the resilience within the community affected by this rare disease, with advocacy groups encouraging it as a time to come together, support one another, and raise awareness about cystic fibrosis (CF). During this month of May, U.S. and…

Pulmonary ionocytes, a rare airway cell type that produces high levels of CFTR — the protein that’s defective in cystic fibrosis (CF) — were found to regulate the acidity of the liquid coating the surface of the airways, a cell-based study showed. Results also showed that club cells, another…

The European Commission (EC) has expanded the approval of Kalydeco (ivacaftor) for the treatment of infants with cystic fibrosis (CF) as young as 1 month with at least one CFTR gating mutation. The approval makes Kalydeco, marketed by Vertex Pharmaceuticals, the first and only CFTR modulator…

Four years after the approval of Trikafta (elexacaftor/tezacaftor/ivacaftor) for cystic fibrosis (CF), global disparities in access to the treatment remain and mainly affect low- and middle-income countries, according to a recent study. “The extent of international disparities, in combination with the high price, suggested that without urgent action,…

Treatment with Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold as Trikafta in the U.S., may save people with cystic fibrosis (CF) an average of 15 minutes each day of respiratory physiotherapy to help with breathing, a study has found. The study, “Perceived burden of respiratory physiotherapy in people with…