Marta Figueiredo, PhD, science writer —

Most people with cystic fibrosis (CF) responding to a U.S.-based online survey were generally satisfied with their treatment plan and were not considering changing their medication. The survey was conducted by Cystic Fibrosis News Today. Shortness of breath was the most commonly reported symptom among CF patients, as well as…

The introduction of CFTR modulators — a type of disease-modifying treatment for cystic fibrosis (CF) caused by specific mutations — has brought a general sense of hope to the lives of young adults with the disease, according to a small single-center U.S. study. Notably, patients reported greater physical…

More than half of children and teens with cystic fibrosis (CF) who do not show breathing difficulties during physical exercise were found to have reduced cardiorespiratory fitness (CRF), a measure of oxygen supply to the muscles, according to a single-center study in the Netherlands. Notably, glucose intolerance and…

The Cystic Fibrosis Foundation (CFF) has appointed Steven Rowe, MD, from the University of Alabama at Birmingham, as its new chief scientific officer. Rowe is the director of the university’s Gregory Fleming James Cystic Fibrosis Research Center and currently holds the Nancy R. and Eugene C. Gwaltney family endowed…

The triple combination therapy Kaftrio restores the function of CFTR — the defective protein in cystic fibrosis (CF) — and associated chloride and bicarbonate flow in gastrointestinal “mini-organs” derived from a patient carrying the most common CF-causing mutation, a study shows. These findings highlight the benefits of Vertex…

Cystic fibrosis (CF) patients show a shift in gut microbiota composition toward more abundant, rarer populations that is significantly associated with antibiotic use, CF disease, and CF-associated changes in gut function and transit, a study shows. These findings add to previous studies showing that CF patients have reduced gut…

Both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan drug status to SPL84-23, SpliSense’s investigational therapy for cystic fibrosis (CF) patients carrying a common disease-causing splicing mutation. The designation is given to treatment candidates with the potential to be safe…

An orally available small molecule called XL147 rescued the function of CFTR — the faulty protein in cystic fibrosis (CF) patients — in lab-grown airway cells from patients carrying F508del, the most common CF-causing mutation, a study shows. Adding XL147 to C18, a compound similar to the CFTR modulator…

One year of Trikafta treatment increases the blood levels of vitamin D in people with cystic fibrosis (CF) who are on vitamin D supplements. These early findings from a small study suggest that the therapy may improve vitamin D supplement absorption, and that vitamin D levels should be…

People with cystic fibrosis (CF) in the U.K. are now living longer, with the life expectancy of children born today surpassing 50 years for the first time, according to a newly published U.K. CF Registry annual data report. The report covered 2020, and the researchers noted: “In this extraordinary year,…