News

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…

Measures of frailty, based on an established index of physical activity, fatigue, weight loss, and the like for older adults, helps in predicting likely outcomes among cystic fibrosis (CF) patients listed for a lung transplant. A greater degree of frailty linked significantly with worsening waitlist status, graft failure, and mortality,…

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 six-week treatment with Kaftrio led to marked improvement in exercise capacity in adolescents with cystic fibrosis (CF), according to an analysis of three cases. The study, “Elexacaftor–Tezacaftor–Ivacaftor improves exercise capacity in adolescents with cystic fibrosis,” was published in the journal Pediatric Pulmology. CF is caused by mutations in…

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…

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…

Allergies to beta-lactam antibiotics were the most commonly confirmed antibiotic allergy in children with cystic fibrosis, according to a small study in Turkey. Sensitivity to multiple types of antibiotics occurred in one-third of children with an allergy, data showed. Having an immediate reaction to a medication and an allergy…

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…

A potential gene-specific therapy for cystic fibrosis (CF) may lie in antisense oligonucleotides, or ASOs, that enhance the production and activity of the truncated CFTR protein caused by the W1282X mutation, a cell study suggested. In combination with approved CFTR modulator therapies, candidate ASOs were seen to raise CFTR-W1282X…