News

Researchers at the University of California, Los Angeles (UCLA) are developing a gene-editing therapy — designed to be delivered as a one-time inhalable treatment — that aims to correct the underlying mutations that cause cystic fibrosis (CF). The team is using tiny fat-based particles to deliver the gene-editing machinery…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to RCT2100, an experimental treatment for cystic fibrosis (CF) that’s currently in early clinical testing for patients who do not respond to or do not tolerate CFTR modulator therapies. The designation is meant to give extra…

Evidence of immune dysfunction — elevated levels of immune cells and immune-related molecules — were found in the lungs of preschool children with cystic fibrosis (CF), even among kids without CF-related lung infections, a new study showed. Treatment with Kalydeco (ivacaftor), but not Orkambi (ivacaftor/lumacaftor), partially restored…

In people with cystic fibrosis (CF) — particularly those with pancreatic insufficiency, which affects digestion — constipation typically led to longer hospital stays and higher healthcare costs, a study found. “Further work is needed to determine management strategies for constipation in hospitalized [people with CF], which may include early…

Poor sleep quality is linked to a higher risk of malnutrition in adolescents with cystic fibrosis (CF), suggesting that sleep disturbances may contribute to added nutritional challenges in these patients, a study in Turkey shows. The effects weren’t influenced by patients’ body mass index (BMI) percentile, a measure of…

Despite often using a contraceptive at some point, unintended pregnancy remains fairly common among women with cystic fibrosis (CF) in the U.S, a survey study found. One in five women reported having an unplanned pregnancy, with the youngest being age 15, adding “to the growing evidence that suggests the…

Discoveries into the process underlying protein organization on cell membranes could ultimately lead to new ways to treat cystic fibrosis (CF), according to a study from Canada that shed light on some of the mechanisms underlying the disease. The study found that proteins linked to CF organize themselves into clusters…

Volatile organic compounds (VOCs) found in breath may be used as biomarkers of lung function and treatment response in children with cystic fibrosis (CF) and mild lung disease, a pilot study in France showed. The study compared breath composition before and up to six months after children initiated Kaftrio…

The Cystic Fibrosis Foundation is once again accepting applications for its Impact Grants, a funding initiative that offers $10,000 per year for up to two years to support programs that empower people living with cystic fibrosis (CF). Applications can be submitted via an online portal and…

The U.K. Medicines and Healthcare Products Regulatory Agency approved Alyftrek (vanzacaftor, tezacaftor, and deutivacaftor) as a cystic fibrosis (CF) treatment for patients ages 6 and older. The triple-combination CFTR modulator, developed by Vertex Pharmaceuticals, is indicated for patients who have at least one copy of a responsive…