Lindsey Shapiro, PhD,  science writer—

CFTR modulator therapy was associated with increases in fecal levels of the pancreatic elastase-1 enzyme (FE-1), reflecting improved pancreatic function, among young cystic fibrosis (CF) patients in a recent study. A small subset of patients achieved FE-1 levels high enough that they were no longer considered to have…

Researchers from Vertex Pharmaceuticals have been awarded the 2024 Breakthrough Prize in Life Sciences for their work developing CFTR modulator therapies for people with cystic fibrosis (CF). Breakthrough Awards, known as the “Oscars of Science,” are granted to scientists who have made important discoveries in life sciences, fundamental…

A European Medicines Agency (EMA) committee has recommended expanding Kaftrio’s (ivacaftor, tezacaftor, and elexacaftor) label to be used in combination with Kalydeco (ivacaftor) in children with cystic fibrosis (CF) ages 2 to 5 who have at least one F508del mutation in the CFTR gene. The CFTR modulator…

A year of treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly reduces recurrent nose and sinus inflammation — called chronic rhinosinusitis, or CRS — in people with cystic fibrosis (CF), though most patients still exhibit signs of severe sinus disease. These are the findings of a new U.S. study that, similar to…

Rates of metabolic and cardiovascular complications — from diabetes and kidney issues to hypertension and abnormal blood-fat levels — increased in the years following a lung transplant among people with cystic fibrosis (CF), according to a recent analysis in the Netherlands. These findings emphasize a need for routine monitoring of…

The community of microbes in the gut, called the gut microbiome, was affected by age, antibiotic use, and geography among infants with cystic fibrosis (CF) in the U.S. and Australia, according to a new study in the two nations. Babies with CF in Australia, where preventive antibiotic usage is…

More than half of the cystic fibrosis (CF) patients who may have been eligible for a second lung transplant died while on a waitlist or had no record of being placed on one, according to a recent analysis in the U.S. and Canada. Among those who did have a…

Bacteriophage therapy could be a promising approach for treating antibiotic-resistant Staphylococcus aureus and Pseudomonas aeruginosa infections in people with cystic fibrosis (CF), according to a small review study. Scientists believe the review, involving three CF patients and two animal models, support moving forward with clinical trials for bacteriophage…

Surrogate markers of nutritional status, including body mass index (BMI) and fat-free mass index, only weakly associated with lung function among children and adolescents with cystic fibrosis (CF) in a recent study. These young patients had different trajectories in nutritional status throughout childhood, with girls tending to maintain a…

Note: This story has been updated Aug. 11, 2023, to correct the definition of a rare disease, which is defined as one affecting fewer than 200,000 people in the U.S., and to reflect that researchers estimate cystic fibrosis may affect about 160,000 people worldwide. Of the more than 100 investigational…