Kalydeco Approved in Europe to Treat Children with R117H Mutation
The European Commission has approved Vertex’s Kalydeco (ivacaftor) for the treatment of children and adolescents with cystic fibrosis (CF) who carry the R117H mutation in the…
The European Commission has approved Vertex’s Kalydeco (ivacaftor) for the treatment of children and adolescents with cystic fibrosis (CF) who carry the R117H mutation in the…
Children with cystic fibrosis (CF) have an altered intestinal virome, or viral communities, compared to healthy children that may impact growth and intestinal inflammation, a case-control study reported. The study, ”The intestinal virome in children with…
Kalydeco treatment led to fewer deaths, transplants, hospitalizations, and exacerbations in people with cystic fibrosis (CF) compared to patients who did not receive the therapy, according to a five-year, real-world analysis of patient registries in the U.S. and U.K. The results of…
People with cystic fibrosis (CF) have higher levels of DNA from common microbes that cause CF-related infections circulating in their blood compared to healthy controls, a study discovered. These findings support the development of a simple blood test as a noninvasive…
Problems with the CFTR protein in platelets, blood cells essential to clotting, activated these cells, both driving lung inflammation and injury, and hindering the lungs’ ability to clear bacteria in a mouse models of cystic fibrosis (CF), a study found. Blocking a calcium channel known as TRPC6 returned platelets…
People with cystic fibrosis (CF) who were treated with Orkambi (lumacaftor/ivacaftor) have significantly less accumulation of fat in the liver — a condition known as hepatic steatosis — which is often associated with CF, a study showed. While hepatic steatosis is common in those…
A triple-antibiotic combination completely eliminated antibiotic-resistant Mycobacterium abscessus, a bacterial infection especially dangerous to people with cystic fibrosis (CF), a study found. Combined doses of three antibiotics — amoxicillin, imipenem, and relebactam — were effective at killing…
Low birth weight and poor weight gain in the first year of life in infants with cystic fibrosis (CF) predict poor growth rates due to pancreatic dysfunction, a study has found. The study, “Factors affecting the growth of infants diagnosed…
Inhibition of the interleukin-1 receptor — to block thick mucus production and inflammation — is a potential strategy to treat cystic fibrosis (CF) in the early stages of the disease before harmful chronic bacterial infections, a study suggests. The study, “…
A biosimilar of dornase alfa inhalation solution, with the same therapeutic activity as Pulmozyme, its reference therapy, was approved for use in Russia, Selexis and Generium Pharmaceutical announced. A…
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