News

Cystic fibrosis (CF) patients and carriers — those with mutations in only one copy of the CFTR, the gene associated with CF — of European ancestry are at a higher risk of developing pneumonia, according to a U.S. study of genetic risk for this lung disease. CF patients were also…

Individuals with cystic fibrosis (CF) from racial and ethnic minority groups in the U.S. are twice as likely to be ineligible for disease-modifying therapies than white patients, because their mutations are not known to be suited to approved treatments, a study reports. The resulting lack of treatment access exacerbates…

The first two patients have been dosed in the Phase 2b OPTION 2 extension study evaluating MS1819 in treating exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF), its developer, AzurRx BioPharma, announced in a press release. The thick mucus that accumulates in the organs of…

Limits placed on social interaction by the COVID‐19 pandemic appear to have led to a drop in the number of pulmonary exacerbations — periods of a sudden worsening in lung health — in children with cystic fibrosis (CF), according to a small U.S. study. This finding — in children ages 2 to…

Researchers boosted the activity of the cystic fibrosis transmembrane regulator (CFTR), the faulty protein at the heart of cystic fibrosis (CF), by altering the levels of a molecule called cyclic AMP (cAMP). Their results suggest that cAMP might make an effective add-on therapy to approved CFTR modulators like…

People with cystic fibrosis (CF) and their families are largely satisfied with the convenience and quality of care received using telehealth models initiated during the COVID-19 pandemic, a survey study from Australia finds. The study, “…

A species of antibiotic-producing bacteria able to inhibit the growth of antibiotic-resistant bacteria linked to chronic lung infections — found in the soils of Northern Ireland and long used in traditional medicine there — have been isolated and shown to have antimicrobial activity, researchers report. The bacteria whose growth they…

The Cystic Fibrosis Foundation (CFF) is awarding Eloxx Pharmaceuticals up to $2 million in additional funding to support the clinical program of ELX-02, a potential therapy for cystic fibrosis (CF) caused by nonsense mutations. “The Cystic Fibrosis Foundation is committed to accelerating treatments for the underlying cause of…

U.K. Rare Diseases Framework, a government-led approach to raising national awareness of rare disorders, accelerating  diagnoses, and improving patient care and treatment, is now in practice. This  framework, signed by all four countries take make up the U.K. — England, Scotland, Wales and Northern Ireland — builds on the success…

The metabolism of cholesterol is impaired in a mouse model of cystic fibrosis, a preliminary study suggests. In the model, there seems to be an altered production of bile salts (those that help with the digestion of fats) that may reduce the digestion and/or absorption of cholesterol. This increases cholesterol’s…