cystic fibrosis

Two new sites for the extension of a Phase 2b trial testing an immediate release capsule form of MS1819 in treating exocrine pancreatic insufficiency in cystic fibrosis (CF) have opened in Poland and are enrolling patients by invitation. Part of the OPTION 2 study (NCT04375878), these European sites join those already open in…

The Cystic Fibrosis Foundation (CFF) has given Kinnear Pharmaceuticals up to $3 million to conduct the laboratory tests of CSA-131, a potential inhaled treatment of infections in people with cystic fibrosis (CF), that are needed to move it into human trials. CF patients are prone to bacterial lung…

The U.S. Food and Drug Administration (FDA) has accepted for review an application to expand the use of Trikafta as a treatment in children with cystic fibrosis (CF) ages 6 to 11 with at least one F508del mutation in the CFTR gene (the gene defective in…

Using bronchoscopy to examine the airways and the surrounding tissues of people with cystic fibrosis, coupled with the collecting of lung fluid through bronchoalveolar lavage (BAL), is safe and a reasonable alternative when sputum samples are not possible, a study reports. Adult patients participating in research reported mild side effects…

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, “…