Steve Bryson, PhD,  science writer—

Researchers have developed nanoparticles that can deliver gene-editing therapeutics to correct genetic defects in the lungs of people with cystic fibrosis (CF). The nanoparticles were optimized using cell-based models to penetrate the thick airway mucus seen in CF. Pretreating patients with the approved mucus-clearing agent Pulmozyme (dornase alfa)…

A University of Iowa biomedical researcher was awarded the Yergin-New International Prize for his work in understanding cystic fibrosis (CF) — with discoveries that helped reveal the mechanisms underlying the genetic condition, and led to the development of a new animal model of CF that captures key features of…

Among methods used to diagnose cystic fibrosis (CF) in France, newborn screening (NBS) was associated with the lowest healthcare expenses during the first year of life, according to a large, population-based study. Fewer inpatient hospitalizations primarily drove the lower costs when infants were diagnosed via NBS, the data showed.

Scientists have discovered a potential therapy for cystic fibrosis (CF) patients who are infected with Mycobacterium abscessus, a bacterium that can cause serious lung infections. They found that a signaling molecule called PI5P can boost the antimicrobial activity of immune cells isolated from patients against the bacterium. This happens even…

A personalized inhaled phage therapy targeting multidrug-resistant lung infections significantly reduces bacterial load and improves lung function in adults with cystic fibrosis (CF), according to a small study. The experimental therapy was personalized by selecting phages that worked best against Pseudomonas aeruginosa strains in patients’ sputum samples. It…

Ivacaftor, a component of the triple-combination therapy Trikafta, is essential to restore mucus clearance in people with cystic fibrosis (CF), a study reports. The study was launched after previous work using CF airway cells suggested that long-term exposure to ivacaftor limited the restoration of CFTR, the protein…

Metered-dose inhalers that use potent greenhouse gases to propel medicines into the lungs of people with cystic fibrosis (CF) have the worst environmental impact across all types of inhalers, according to a new study from researchers in Spain. The team noted that propellants in metered-dose inhalers are hydrofluorocarbons, potent…

At least three months of Kaftrio treatment reduced liver stiffness, an indicator of liver fibrosis, in school-age children and adolescents with cystic fibrosis (CF), according to a real-world study in Sweden. The benefits were particularly prominent among children who had signs of CF-related liver disease (CFLD) before starting Kaftrio,…

Long-term Trikafta treatment led to improved lung function, fewer respiratory symptoms, and lower sweat chloride levels — elevated levels are a key hallmark of cystic fibrosis (CF) — in children with CF, ages 6 to 11, according to new findings from a two-year extension study. Trikafta was…

The European Commission has approved a label expansion for Kaftrio in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) caused by a wider range of mutations. The decision follows a positive endorsement by the Committee for Medicinal Products for Human Use, a branch of the…