All babies with a known mutation causing cystic fibrosis (CF) and a second mutation known as the 5T allele should be screened for additional mutations to predict their risk of developing CF later in life, according to a study conducted by researchers at Children’s Hospital Los Angeles (CHLA), Brigham and Women’s Hospital (BWH), and the California Department of Public Health.
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The Cystic Fibrosis Foundation hosted more than 100 researchers from various fields at its “New Technologies Advancing Toward a One-time Cure” conference last month to debate how new research and treatments for genetic diseases across the board could lead to a cure for cystic fibrosis (CF), an inherited condition in which the lungs and digestive system can…
When patients with cystic fibrosis (CF) breathe in small amounts of bile, some bacterial species switch to a chronic infection mode, aiding both bacterial colonization of the airways and the development of antibiotic resistance. The study, “Bile signalling promotes chronic respiratory infections and antibiotic tolerance,” published in the…
Suppressing a specific type of immune cell called Th17 may aid cystic fibrosis (CF) patients with Pseudomonas aeruginosa infection, one of the most common infections in CF, as suggested by findings of a recent study in mice. The study, “Antiinflammatory effects of bromodomain and extraterminal domain inhibition in cystic…
Patients and loved ones affected by the burden of cystic fibrosis (CF) may want to take action and do something to fight this chronic and progressive disease. However, it’s not always easy to find ways to do so. Becoming an advocate for cystic fibrosis may be an option for some…
ProQR Therapeutics announced that QR-010, an inhaled therapy it is developing to treat cystic fibrosis patients with the ∆F508 mutation in the CFTR gene (the gene that is defective in CF), has received Fast Track designation from the U.S. Food and Drug Administration (FDA) to speed its testing and review. The drug is now being…
Platelets circulating in the blood of cystic fibrosis (CF) patients do not contribute to the increased levels of pro-inflammatory cytokines known to exist in these patients, and which aggravate the chronic inflammation that marks the disease, researchers reported. Their study, “Decreased TGF-β1 and VEGF Release in Cystic Fibrosis Platelets: Further Evidence for Platelet…
Savara Pharmaceuticals announced that it has finished with the steps necessary to fully acquire Serendex Pharmaceuticals, strengthening its position as a developer of therapies for rare respiratory diseases. Savara’s pipeline of products now includes these three, according to a press release: Alveodex, a proprietary nebulized formulation of Factor VIIa (recombinant human FVIIa),…
Pharmaxis announced that it has completed recruiting adult patients with cystic fibrosis (CF) for its large and potentially pivotal Phase 3 clinical trial evaluating Bronchitol (mannitol) as an inhaled treatment. The company expects to report results in the second quarter of 2017. Based on those results, the company will apply for U.S. Food and Drug…
Interleukin-22 is known to protect against respiratory pathogens through its ability to mediate mucosal immunity and tissue regeneration. But new research into its possible use against Pseudomonas aeruginosa, a main cystic fibrosis pathogen, found that IL-22 disappointed — with scientists reporting IL-22 failed to protect a mice model from such infection and even seem to…
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