Patricia Inácio, PhD,  science writer—

Genetic screening for cystic fibrosis mutations helps individuals and couples identify their risk of having a child with the disease, an Australian study shows. The research, published in the journal Genetics in Medicine, is titled “Reproductive genetic carrier screening for cystic fibrosis, fragile X syndrome, and spinal…

The benefits of current therapies to treat cystic fibrosis (CF) are limited, so scientists are exploring new strategies based on increasing the expression and activity of alternative chloride channels, such as the ANO1 channel. In a new study, researchers have advanced a potential therapeutic strategy to restore cells’ normal functioning…

European researchers have identified a group of genes that behave as potential modifiers in cystic fibrosis (CF) patients with a F508del mutation in the CFTR gene — a discovery that may lead to new ways to treat the disease. Their study, “Transcriptomic profile of cystic fibrosis patients identifies type I…

AzurRx BioPharma has enrolled the sixth patient in a Phase 2 clinical trial of MS1819 as a treatment for a digestive disorder known as exocrine pancreatic insufficiency that is associated with cystic fibrosis. The company hopes to enroll 12-15 patients in the study (ACTRN12616000962437), which it is conducting in Australia and…

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have accepted for priority review applications for a combination of tezacaftor and Kalydeco (ivacaftor) as treatment for a subset of cystic fibrosis (CF) patients, Vertex Pharmaceuticals announced. “If approved, the tezacaftor/ivacaftor combination treatment would become Vertex’s third medicine to treat the underlying cause of…

Inhalation of hypertonic saline solution (HSS) by children with cystic fibrosis (CF) increased sputum production and improved pathogen identification. These results suggest that this simple procedure, if implemented routinely in doctors’ offices and clinics, would help patients who can’t secrete mucus spontaneously. The study, “Hypertonic Saline as a Useful Tool…

Verona Pharma recently announced the company’s accomplishments in the last six months, including an update to the company’s clinical trial in cystic fibrosis (CF) and positive interim results for its investigational therapy RPL554. RPL554 is an inhaled dual inhibitor of two enzymes, phosphodiesterase 3 and 4, and was previously shown to…

The U.S. Food and Drug Administration (FDA) is advising patients and healthcare professionals to not use any liquid products made by PharmaTech, LLC due to possible contamination with Burkholderia cepacia bacteria, which may cause serious infections in patients with chronic lung diseases, including cystic fibrosis (CF). The regulatory agency previously had…

Concert Pharmaceuticals’ sale of its cystic fibrosis therapy CTP-656 to the larger Vertex Pharmaceuticals will lead to cystic fibrosis patients obtaining faster access to the therapy, Concert CEO Roger Tung predicted. He also said that the $160 million that Concert obtained from the sale will allow the company to advance its hair-loss therapy alopecia areata CTP-543 into…

Sweat from infants with cystic fibrosis contains substances that could be used as biomarkers of the disease and even identify new disease mechanisms, a study reports. The research, published in the journal ACS Central Science, was titled “The Sweat Metabolome of Screen-Positive Cystic Fibrosis Infants: Revealing Mechanisms beyond Impaired Chloride Transport.” Scientists…