News

LGG Supplement May Ease CF By Changing Gut Microbiome

Children with cystic fibrosis (CF) whose intestinal microbe populations — or gut microbiome — were dominated by Bifidobacteria had fewer hospitalizations and pulmonary exacerbations, needed fewer antibiotics, and showed lower inflammation in the gut than children with a gut microbiota favoring Bacteroides, a study reported. A dominance of Bifidobacteria…

eRF3a Protein May Be Target in Treating CF Nonsense Mutations

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…

Most Adults in CF Study Have Low Bone Mineral Density

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…

Phase 1 Trial for CF Gene Therapy KB407 to Open in US

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…

Reducing Treatment Burden Key to CF Patients, Survey Suggests

Adults with cystic fibrosis (CF) are willing to accept reductions in lung function and life expectancy as a trade-off for a reduced treatment burden, a survey of patient preferences suggested. Over all other outcomes, the participants preferred a treatment choice that would extend their life expectancy by 10 years…

Mothers of CF Children Need Support for Caregiver Burnout: Study

Mothers who care for children with cystic fibrosis (CF) often experience depression, burnout, caregiver burden, and negative attitudes toward their children, a study suggests. These caregiving struggles may increase with more hospital visits and hospitalizations, and having a lower family income, more children, and another child with a chronic…