News

The Phase 1 clinical trial into the safety and pharmacological properties of the antibiotic Xenleta (lefamulin) in adults with cystic fibrosis (CF) is fully enrolled. “We are happy to announce enrollment has been completed in this important study to evaluate the potential of XENLETA in the management of…

Treatment with the triple-combination therapy Trikafta improved lung function and eased respiratory symptoms for school-aged children with cystic fibrosis (CF) in a Phase 3 clinical trial. “I was both surprised and delighted to see that, even this early in the disease trajectory and despite the brief treatment duration,…

Efforts to promote physical activity in people with cystic fibrosis (CF) should be holistically integrated into routine care, rather than delivered as separate interventions. “The key message resulting from the current research is that the promotion of [physical activity] in this population would be most effective as part of…

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…

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…