Margarida Maia, PhD,  science writer—

Body composition assessments, particularly fat mass and fat-free mass, may be more informative than body mass index (BMI) for monitoring weight gain in cystic fibrosis (CF) patients starting Kaftrio (elexacaftor/tezacaftor/ivacaftor), a Welsh real-world study suggests. The researchers found that while patients’ BMI, a ratio of weight to height, significantly…

First Wave BioPharma has finished dosing in its Phase 2 clinical study, SPAN, in which a new adrulipase formulation is being tested as a potential exocrine pancreatic insufficiency (EPI) treatment in people with cystic fibrosis (CF). With the last patient dosed, the company remains on track for next…

Kaftrio improved lung function for up to 2.5 years in cystic fibrosis (CF) patients with advanced disease who had early access to the medication in Spain, according to a real-world study. Taking Kaftrio, a triple combination of elexacaftor, tezacaftor, and ivacaftor, also reduced the frequency of exacerbations, when…

Nearly half of patients with cystic fibrosis (CF) due to a G542X mutation saw their lung function improve with ELX-02 combined with Kalydeco (ivacaftor), according to final data from a Phase 2 clinical trial. Top-line data released last September suggested ELX-02, an experimental medicine by Eloxx…

Taking Kaftrio, a combination of elexacaftor, tezacaftor, and ivacaftor sold as Trikafta in the U.S., for up to 24 weeks can make signs of inflammation go down in people with cystic fibrosis (CF), and help get rid of Pseudomonas aeruginosa bacteria. That’s according to data from a…

Children with cystic fibrosis (CF) who also have symptoms of attention-deficit/hyperactivity disorder (ADHD) may experience worse lung function, according to a new study from Israel and Spain. But the study also spotlighted a potentially far-reaching impact from ADHD on CF symptoms and care for children and adults…

Vertex Pharmaceuticals has received a positive opinion from an arm of the European Medicines Agency on expanding the use of Orkambi (lumacaftor/ivacaftor) to children ages 1-2 who have two copies of the F508del mutation, the most common genetic defect in cystic fibrosis (CF). With the recommendation from…

Communities of fungi in the airways can be more diverse in cystic fibrosis (CF) patients with better lung function and those using medications known as CFTR modulators, a study found. “As CF lung disease progresses, bacterial richness and diversity are known to decrease, concomitant with increased absolute and relative…

Overactive T2 inflammation, a type of immune response linked to asthma, may be associated with worse symptoms of cystic fibrosis (CF), a finding that could pave the way for using anti-asthmatics for CF, a study suggests. The study, “Association between Cystic Fibrosis exacerbations, lung function, T2 inflammation and…

Use of Kaftrio (elexacaftor/tezacaftor/ivacaftor) may help people with cystic fibrosis (CF) and the F508del mutation in both CFTR gene copies live to a median age of 71.6 and possibly longer if treatment is started in adolescence, a U.K.-based study reported. In a scenario where treatment with Kaftrio — sold…