cystic fibrosis

Problems with a protein called TAS2R38 are linked to a greater risk of severe sinonasal disease and Pseudomonas aeruginosa lung infections early in life in people with cystic fibrosis (CF), a study reported. These findings may help to identify high-risk patients, those with a dysfunctional form of TAS2R38, and improve approaches…

Delivering nutrients through dialysis could help people with co-occurring cystic fibrosis (CF) and kidney disease meet their nutritional requirements, a recent case report illustrates. The report, “Intradialytic parenteral nutrition improves nutritional status in a complex cystic fibrosis patient with redo double lung transplant and end-stage renal…

Proteostasis Therapeutics reported receiving scientific advice for its CHOICES clinical trial program, which is to test its personalized triple combination for cystic fibrosis (CF) patients without available treatment options, from the Dutch Medicines Evaluation Board. Regulatory officials outlined necessary steps in this planned, pivotal trial that would support…

Arrowhead Pharmaceuticals has filed for approval to launch a Phase 1/2a clinical trial in New Zealand testing its candidate inhaled therapy for cystic fibrosis (CF), called ARO-ENaC, the company announced. ARO-ENaC is an investigational RNA therapy designed to lower the production of the epithelial sodium channel alpha subunit (αENaC) in the…

Enrollment is continuing in a Phase 1/2 trial testing the safety and properties of AR-501, a potential inhalation treatment for chronic lung infections associated with cystic fibrosis (CF), Aridis Pharmaceuticals announced. The company also remains on track during the COVID-19 pandemic to report top-line trial findings: those in healthy volunteers…

Most healthcare professionals are not fully prepared for cystic fibrosis (CF) end-of-life care, and further training and coordination between CF and palliative care teams would be helpful, according to a small, single-center survey in the U.K. Its researchers recommended a broader survey — one nationwide, or even international — to…

An unusual clinical presentation can hinder proper diagnosis for people with cystic fibrosis (CF), even with newborn screening, a new case report contends. The report, “Uncommon clinical presentation of cystic fibrosis in a patient homozygous for a rare CFTR mutation: a case report,” was published in…