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Dosing has begun in a Phase 1 clinical trial of CM001, an inhaled treatment designed to act as a molecular prosthetic in cells and treat cystic fibrosis (CF) regardless of a patient’s underlying mutation, Cystetic Medicines, the therapy’s developer, announced. Most immediately, the company expects that CM001…

Trikafta treatment for six months in people with cystic fibrosis (CF) was found in a real-world study to reduce the thickness of the airway walls and lead to less excessive mucus plugging in the lungs. Overall, as observed by MRI, the approved CF therapy improved structural abnormalities in…

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…

In the months after starting on Trikafta, people with cystic fibrosis (CF) generally experience lower rates of hospitalizations and lung infections — but nonetheless, the total cost of healthcare for these patients tends to increase, a new economic analysis indicates. The findings are in line with prior analyses…

A1c, a test that measures the average levels of sugar in the bloodstream, was able to predict the future risk of adult and pediatric cystic fibrosis-related diabetes (CFRD), a study shows. Elevated CFRD risk was associated with an A1c value of 5.5% or above in children and 6% and…

Children with cystic fibrosis (CF) who have normal pancreas function — called pancreatic sufficiency (PS) — are at higher risk of being overweight and obese than those with pancreatic insufficiency (PI), according to a single-center study in Greece. According to its researchers, findings support a reevaluation of “the recommendation…

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…

Top-line results are expected next month for the SPAN clinical trial, a Phase 2 study testing First Wave BioPharma‘s new formulation of adrulipase to treat exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). The trial’s target enrollment has been reached, with 13 total participants signed on,…

Long-term treatment with Trikafta leads to sustained improvements in lung function, easing of respiratory symptoms, and better nutritional status for people with cystic fibrosis (CF) who have at least one copy of the F508del mutation. That’s according to new data presented by the therapy’s developer Vertex Pharmaceuticals…

Rates of screening for diabetes among people with cystic fibrosis (CF) are not where they should be, a new study reports. “Studies to improve our approach to [CF-related diabetes] screening are urgently needed,” the researchers wrote in “A provider survey of cystic fibrosis related diabetes screening and…