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…
Blood levels of two hormones, kisspeptin and proopiomelanocortin, are lower in people with cystic fibrosis (CF) — beginning in childhood and continuing through adulthood — than in healthy people, a study found. These reduced concentrations may be associated with lower body weight, delayed puberty and poorer fertility in CF…
Twelve months into the COVID-19 pandemic, Australian adults and children with cystic fibrosis (CF) showed improved lung function and body mass index (BMI), as well as a lower hospitalization rate, a study reports. “Differing models of care and major changes in community interactions both have the…
Differences in levels of proteins related to wound healing, cell migration and neurological development may influence whether or not people with cystic fibrosis (CF) experience improved lung function with Kalydeco (ivacaftor), according to a new analysis. The analysis found that “differences in…
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