Marisa Wexler, MS,  senior science writer—

Targeting a protein called eRF3a may offer a way of treating cystic fibrosis (CF) caused by nonsense mutations, potentially opening a treatment avenue to people whose rare disease-causing mutations do not respond to current CFTR modulators, researchers reported. Their study, “Small molecule eRF3a degraders rescue…

More than half of adults with cystic fibrosis (CF) who underwent assessments of bone mineral density at a U.S. center had lower-than-expected bone density, a new study reports. “The prevalence of CF adults with lower than expected BMD [bone mineral density] in this study was found to be more…

Among babies with cystic fibrosis (CF) identified via newborn screening, there are marked delays in the start of care for children from racial and ethnic backgrounds that have historically been disadvantaged, according to a new study done in the U.S. “Because pre-symptomatic treatment is the overarching goal of [newborn…

A Phase 1 clinical trial of an experimental inhalation gene therapy for cystic fibrosis (CF) — KB407 by Krystal Biotech — was given clearance to start by the U.S. Food and Drug Administration (FDA). Expected to begin this year, the study aims to enroll 20 adults with CF who…

Infusions with a hormone called GLP-1 boosted insulin production in cystic fibrosis (CF) patients with impaired glucose tolerance, according to data from a small study. The results suggest that GLP-1 agonists — medications that mimic the hormone’s activity, but last longer in the body — may be useful as…

People with cystic fibrosis (CF) who use inhaled antibiotics are at more than three times higher risk of becoming infected with the fungus Aspergillus fumigatus, according to a new study. Study results showed no association between Aspergillus infection and lung function decline, and available data demonstrated no clear benefits of treating the…

Treatment with Kalydeco (ivacaftor) significantly reduces the levels of inflammatory molecules in the lungs of preschool-age children with cystic fibrosis (CF), according to a small study. A reduction in lung inflammation was not observed in preschoolers with CF who were treated with Orkambi (lumacaftor/ivacaftor). These findings add…

The government of Ontario has expanded coverage of Vertex Pharmaceuticals’ triple-combination treatment Trikafta for children, 6 and older, with cystic fibrosis (CF) and at least one copy of the most common CF-causing mutation, called F508del. The announcement comes a few months after Health Canada expanded Trikafta’s previous label,…

A technique called 2D shear wave elastography, which measures the stiffness of tissues, could be useful for detecting liver disease in people with cystic fibrosis. That’s according to the study, “2D Shear Wave Elastography, a promising screening tool for Cystic Fibrosis liver disease, shows a correlation…

Treatment with the probiotic Vivomixx appeared to lead to healthful changes in the gut microbiome of cystic fibrosis (CF) patients in a small clinical trial, including six with CF-related…