News

The nonprofit Emily’s Entourage has awarded $220,000 to a project to develop a nonviral and inhalable gene therapy for cystic fibrosis (CF). The grant was awarded to Lorraine Martin, PhD, professor at Queen’s University Belfast, U.K. Martin is developing the therapy in collaboration with OmniSpirant Therapeutics. The…

Nearly two-thirds of the people with cystic fibrosis (CF) in the U.S. are under financial hardship, and many face unmet medical needs, meaning their treatment is not being addressed adequately. Researchers with George Washington University and the Cystic Fibrosis Foundation, who conducted this analysis into the cost of…

In-school use of airway clearance therapy (ACT) improved a number of health outcomes for children with cystic fibrosis (CF) who had poor adherence to ACT at home, according to a new study. In the year after starting ACT at school, the children experienced significant reductions in the number…

Type 2 diabetes in a 49-year-old woman with cystic fibrosis (CF) went into remission after bariatric surgery, given to help her lose weight after obesity was seen as the likely cause of her blood-sugar levels. She had a mild form of CF, stable pulmonary function and pancreatic sufficiency, supporting…

Taking Trikafta (elexacaftor/tezacaftor/ivacaftor) may lead to only a mild rise in liver enzymes in people with cystic fibrosis (CF)-related liver disease, a study found. This is likely not a clinically significant change, “and should not be a barrier to prescription,” the researchers wrote in the study, “Longitudinal effects…

A Phase 3 clinical trial called COPILOT, which will test the antibiotic ColiFin (inhaled colistimethate sodium) in people with cystic fibrosis (CF) who have chronic Pseudomonas aeruginosa lung infections, is expected to begin later this year. The trial is expected to enroll 38 adults and adolescents with moderate…

BreatheCon 2023 — the seventh annual virtual gathering offering online support for adults with cystic fibrosis (CF) — is taking place this year from Feb. 23-25. Registration for the event, hosted by the Cystic Fibrosis Foundation, is now available on the program’s webpage. The focus of the…

A Phase 2 clinical trial of an optimized formulation of adrulipase, a yeast-derived enzyme to treat exocrine pancreatic insufficiency (EPI) in adults with cystic fibrosis (CF), has screened its first patient. First Wave BioPharma, the therapy’s developer, anticipates dosing to start soon, with top-line results expected by mid-year.

Nearly 8% of people with cystic fibrosis (CF) may be infected with nontuberculous mycobacteria (NTM), a type of bacteria in the environment, a meta-analysis study has found. Researchers observed that the number of cases has surged across the world over the last few years. The bacteria can cause infection…

A Phase 1 trial of ARCT-032, an inhaled experimental treatment for cystic fibrosis (CF), has been cleared to start in New Zealand. Developed by Arcturus Therapeutics, ARCT-032 contains the messenger RNA (mRNA) that provides instructions for lung cells to produce the CFTR protein, restoring its activity. Mutations in…