More lumps of fat and hardened deposits, called calcifications, may show up on imaging scans of the pancreas of people with cystic fibrosis (CF) who have diabetes, a study shows. These findings suggest doctors can use computed tomography (CT) imaging to help diagnose CF-related diabetes and distinguish these…
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Being treated with Kaftrio or Symveki led to a reduced need for insulin in people with cystic fibrosis-related diabetes (CFRD), a small study found. The treatment wasn’t associated with alterations in blood sugar and associated factors in CF patients without diabetes. The findings add to a growing…
People with cystic fibrosis (CF) who were underweight before treatment gained the most weight after six months on Trikafta (elexacaftor/tezacaftor/ivacaftor), according to a new study. Underweight participants, as assessed with the body mass index (BMI), also saw the biggest gains in treatment-related lung function. Patients who were overweight…
Kaftrio, sold as Trikafta in the U.S., continues to thin mucus and to reduce the number of harmful bacteria in the airways when taken for over a year by people with cystic fibrosis (CF), according to an observational study in Germany. While long-term use of Vertex…
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…
The European Commission approved expanding the use of Orkambi (lumacaftor/ivacaftor) to include children as young as 1 year old with cystic fibrosis (CF) and two copies of the F508del mutation in the CFTR gene. Vertex Pharmaceuticals’ therapy was first approved in the European Union…
The first patient has been dosed in a Phase 1 clinical trial of KB407, Krystal Biotech’s investigational gene therapy for people with cystic fibrosis (CF) regardless of the type of disease-causing CFTR gene mutation. The Phase 1 CORAL-1/US study (NCT05504837) is recruiting up to 20 adults with…
The presence of intestinal metabolites called bile acids in the airways of 1-year-old children genetically diagnosed with cystic fibrosis (CF) is significantly associated with early disease mechanisms and worse clinical outcomes, according to a study in Australia. These include increased levels of airway inflammation biomarkers, greater structural lung damage,…
Treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) can improve lung function and nutritional status for people with cystic fibrosis (CF) caused by the N1303K mutation, a small real-world study reports. The N1303K mutation is not among the 178 disease-causing mutations in the CFTR gene considered to be responsive to Trikafta and…
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…
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