An experimental RNA-based approach restored production and function of CFTR — the protein that is faulty or missing in cystic fibrosis (CF) — in preclinical models carrying disease-causing nonsense mutations, a study shows. These mutations introduce an incorrect stop signal in the genetic instructions for making CFTR, causing cells…
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Bacterial extracellular vesicles (bEVs) — tiny sacs released by bacteria containing pro-inflammatory molecules — have been found at elevated levels in the bloodstream of people with cystic fibrosis (CF), particularly in those whose lungs are infected with the bacterium Pseudomonas aeruginosa. In the pilot study, the researchers call…
A single dose of an inhaled experimental gene therapy for cystic fibrosis (CF) — one designed to treat people regardless of their disease-causing genetic mutation — was shown in an animal model to sustainably boost CFTR gene activity and protein levels in the lungs and spread to several other…
Two U.S.-based researchers aiming to help gene-corrected airway stem cells grow and survive long enough to repair lung tissue — with the goal of developing a long-lasting treatment for cystic fibrosis (CF) — have received a grant from the nonprofit Emily’s Entourage. This type of stem cell therapy may…
A study found machine learning models could help predict whether chronic lung infections in people with cystic fibrosis (CF) will be resistant to commonly used antibiotics, helping doctors make more informed treatment choices. By linking a decade of information on patients’ antibiotic resistance with previous antibiotic use and other…
Developing cystic fibrosis (CF) during the first month of life may no longer necessarily predict poorer long-term outcomes, a new study from Portugal suggests. Despite experiencing substantial disease burden early in life, people whose signs of CF first manifested during the neonatal period had similar long-term nutritional outcomes and survival…
Writer, director, and actor Leah McKendrick has been selected as the winner of the 2026 Clairity Spotlight Award in recognition of her work raising awareness of cystic fibrosis (CF). McKendrick is the creator of the Netflix romantic comedy “Voicemails for Isabelle.” This award is given each year by…
Continued treatment with CFTR modulators was linked to lower anxiety and depression scores in children with cystic fibrosis (CF) along with improvements in lung function and parents’ mental health and quality of life, a study found. The study, “Mental health, sleep, and quality of life following CFTR…
Trikafta (elexacaftor/tezacaftor/ivacaftor) was associated with improved lung function and fewer pulmonary exacerbations, or sudden worsening of lung symptoms, in children ages 6 to 11 with cystic fibrosis (CF), a real-world study found. The treatment was also associated with improved nutritional status, fewer hospitalizations and antibiotic treatments, and reduced…
A Phase 2a clinical trial testing RCT2100, Recode Therapeutics’ investigational inhaled mRNA therapy for cystic fibrosis (CF), is fully enrolled, with results expected by the end of the year, the company announced. The clinical updates were part of a broader company announcement focused on Recode’s CF programs, which also…
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