News

Treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) contributed to a reduction in pulmonary exacerbations requiring hospitalization between 2018 and 2022 among children and young adults with cystic fibrosis (CF). That’s according to a recent study in the U.S. that also demonstrated that the proportion of hospitalized CF patients from minority groups,…

Pregnancy does not negatively impact survival or lung function in women with cystic fibrosis (CF) and, in fact, may be associated with a lower risk of death, according to a U.S. study that compared CF women who had been pregnant with those who had never been pregnant. No significant…

The European Commission has approved a label expansion for Kaftrio in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) caused by a wider range of mutations. The decision follows a positive endorsement by the Committee for Medicinal Products for Human Use, a branch of the…

Registration is now open for ResearchCon 2025, a free event the Cystic Fibrosis Foundation assembles each year for researchers, doctors, and patients to share and discuss the latest advances in research and clinical care in cystic fibrosis (CF). This virtual event, set online for May 6, provides…

The Cystic Fibrosis Foundation (CFF) has published updated newborn screening (NBS) guidelines to help ensure better equity in the diagnosis and treatment of cystic fibrosis (CF) in the U.S. A key recommendation is that, should an initial NBS result be positive, follow-up genetic testing should involve all possible…

Trikafta (elexacaftor/tezacaftor/ivacaftor) improves lung function and nutrition status in adults and adolescents with cystic fibrosis (CF), and appears to work particularly well in those with more severe lung disease, according to a real-world study from the U.S. Researchers also observed that Trikafta appears to work about as well…

The Cystic Fibrosis Foundation will invest up to $2.3 million for a company to develop a breath test designed to detect Pseudomonas aeruginosa and to monitor chronic infections in people with cystic fibrosis (CF). P. aeruginosa bacteria are a major cause of lung disease in people with CF. Although…

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…