Marisa Wexler, MS,  senior science writer—

In a small clinical trial, Orkambi (lumacaftor/ivacaftor) appeared to reduce structural damage in the lungs, as measured by a chest MRI, for preschool-age children with cystic fibrosis (CF) who are homozygous for the F508del mutation. “This study suggests that [Orkambi] may modify CF disease progression when administered early…

Packets of molecular cargo called extracellular vesicles (EVs) can be isolated from the blood of people with cystic fibrosis (CF) and their contents may differ from those who don’t have the disease. The findings also suggest these contents in CF may be changed by CFTR modulator therapy and…

A new company, Inspire Biotherapeutics, has launched with the goal of developing a new gene therapy platform that could be used in the treatment of cystic fibrosis (CF) and other disorders that affect the lungs. Inspire is planning to launch a first-in-human clinical trial to test the novel…

AR-501, an experimental, nonantibiotic inhaled treatment for chronic lung infections in cystic fibrosis (CF), was well tolerated among adults patients in a Phase 2a clinical trial. Top-line data, announced by the therapy’s developer Aridis Pharmaceuticals, suggest that the medication was present in lung fluid samples at concentrations…

The IRT test, commonly used for screening newborn for cystic fibrosis (CF), is most accurate when performed between three to six days after at-term babies have been born, a study shows. A repeat IRT test may be diagnostically useful in certain cases, such as for very pre-term babies,…

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…

Among children with cystic fibrosis (CF), bone health tends to become worse with older age — and poor bone health in CF is linked to less activity of nearby muscle — a new study suggests. The findings imply that promoting physical activity for children with CF could help to…

Having cystic fibrosis (CF) can put a tremendous financial strain on patients: In the U.K., the typical family affected by the genetic disease will lose more than £6,500 (about $8,000) every year as a consequence. That’s according to a new report, “The Financial Costs of Cystic…

The Cystic Fibrosis Foundation is investing up to $15 million in ReCode Therapeutics to support the development of the company’s inhaled mRNA-based therapy for cystic fibrosis (CF). “We are excited to expand our relationship with the CF Foundation with this investment in our CFTR mRNA program for cystic…