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…
News
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…
Increasing the pH balance of the acidic lung surface liquid found in cystic fibrosis (CF) patients, using an inhaled alkaline buffer, helps to kill bacteria in the airways, research from the University of Iowa Carver College of Medicine found. The study, “Repurposing tromethamine as inhaled therapy to treat…
Adult patients with cystic fibrosis (CF) show improvements in lung function decline when treated with a high dosage of tiotropium bromide taken over a long term (24 months). Patients with cystic fibrosis often succumb to chronic lung disease, the leading cause of death among cystic fibrosis patients. Chronic lung disease is often…
Vertex Pharmaceuticals and Moderna Therapeutics recently entered a three-year research partnership and licensing agreement to discover and develop the messenger ribonucleic acid, trademarked as (mRNA) Therapeutics, for the treatment of cystic fibrosis (CF). The alliance between the two companies will center on usimg mRNA drugs to treat the CF  at its core, by…
Nivalis Therapeutics Inc. recently announced that they finished enrolling participants for a Phase 2 clinical trial testing their lead compound N91115 for the treatment of cystic fibrosis (CF).
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