A novel gene-editing tool that allows scientists to “drag-and-drop” sequences of DNA into the genome, without inducing large cuts, could aid in treating genetic diseases like cystic fibrosis (CF), a study reported. The technology, aptly called PASTE (for programmable addition via site-specific targeting elements), “expands the capabilities of genome…
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Enrollment of adults at sites across the U.S. is continuing for a Phase 2a trial of AR-501 (gallium citrate), Aridis Pharmaceuticals‘ investigational inhaled therapy for chronic lung infections due to cystic fibrosis (CF). As an inhaled treatment, AR-501 is intended to be self-administered weekly using a hand-held nebulizer that…
A year of insulin treatment improves nutritional status and respiratory function in cystic fibrosis (CF) patients with diabetes, according to a study in Italy. In addition, CF patients treated with insulin at an early stage of abnormal blood sugar levels have reduced hospital admissions due to…
Vertex Pharmaceuticals recently filed an application with the U.S. Food and Drug Administration (FDA) asking that Trikafta, its triple-combination modulator therapy, be approved for children with cystic fibrosis (CF) ages 2 to 5. The company is planning to file similar requests in the European Union and U.K.
The Cold Spring Harbor Laboratory (CSHL) has licensed an artificial intelligence platform, developed by Epistemic AI, to accelerate cystic fibrosis (CF)-related discoveries. The platform, which also contains CF-specific resources, will help the lab’s scientists to accelerate their research by providing easy access to publications and clinical trials,…
The rate of new cases, or incidence of cystic fibrosis (CF) is gradually and annually decreasing in the U.S. and Canada alike — and has been for nearly a quarter of century — a new study found. From 1995 to 2019, CF incidence has dropped at a rate of…
Most people with cystic fibrosis (CF) have fungi in their airways, but the fungi may come and go in waves of activity, a Spanish study suggests. The study, “Fungal microbiota dynamics and its geographical, age and gender variability in patients with cystic fibrosis,” was published in Clinical Microbiology…
Adults and children with cystic fibrosis (CF) frequently have moderate to severe gastrointestinal (GI) symptoms such as fullness and bloating, and women are primarily affected, new research suggests. GI-targeted medications were largely not effective and both age groups reported being highly dissatisfied with them. The study, “Multicentre prospective…
A new test that measures the excretion of bicarbonate in urine may be used to assess the effectiveness of treatments for cystic fibrosis (CF), according to a new study. “The goal is for the urine test to be used as a clinical tool to determine both the severity of…
Defects in both epithelial and immune cells contribute to changes in the gut microbiota, the populations of microbes naturally present in the gut, seen in cystic fibrosis (CF), a recent study in mice showed. While the immune component had the greater effect in the small intestine, however, the epithelial…
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