Joana Carvalho, PhD, managing science editor —

A single dose of MRT5005, Translate Bio‘s first-in-class messenger RNA (mRNA) therapy for the treatment of cystic fibrosis (CF), improves patients’ lung function, interim data from a Phase 1/2 trial shows. While stressing these are early results, the researchers called the findings “promising.” CF is a…

The European Commission (EC) and the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) granted Mylan the right to market TOBI Podhaler (tobramycin inhalation powder) and TOBI Solution (tobramycin nebulizer solution) in the United Kingdom, the company announced. The decision came after the Committee…

Sigrid Ladores, PhD, an associate professor at the University of Alabama at Birmingham School of Nursing, was awarded a $150,000 two-year grant by the National Institutes of Health (NIH) to support research focused on preserving fertility in women with cystic fibrosis (CF) who are likely candidates for…

Oleh Taratula, PhD, a researcher at Oregon State University‘s College of Pharmacy, has been awarded a $2.3 million five-year grant to support a research project into a muscle-wasting disorder called cachexia that can affect people with chronic diseases that range from cancer to cystic fibrosis, multiple sclerosis, Crohn’s,…

Eluforsen, an investigational therapy for the treatment of cystic fibrosis (CF), improves the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in cellular and mice models of CF that carry the most common mutation associated with the disorder, a study says. The findings of the study,…

People with cystic fibrosis (CF) may require a modified and more rigorous cleansing procedure to prepare their bowels for a colonoscopy screening procedure that allows good visualization, a study reports. Findings of the study, “A non-randomised single centre cohort study, comparing standard and modified bowel preparations, in adults with…

The U.S. Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) tablets for the treatment of children, ages 6 to 11 years, with cystic fibrosis (CF) who have certain mutations that respond to treatment, its developer, Vertex Pharmaceuticals, has announced. In order…

Small proteins with antimicrobial properties found in the mucus of the brown garden snail may open the door to the development of new treatments for patients with cystic fibrosis (CF) who often experience lung infections caused by the bacteria Pseudomonas aeruginosa, a study says. The findings of the…

EffRx Pharmaceuticals announced that it has signed an exclusive license agreement with Pharmaxis to register and commercialize Bronchitol (mannitol) for the treatment of cystic fibrosis (CF) in Switzerland. According to EffRx, Bronchitol should be available to Swiss patients by 2021. Bronchitol is a dry powder for…

Murepavadin (POL7080), Polyphor‘s lead investigational antibiotic targeting Gram-negative bacteria, behaved as expected and was well-tolerated when administered to mice harboring a lung infection caused by Pseudomonas aeruginosa, data from a preclinical study show. Of note, P. aeruginosa is a bacteria that often infects the lungs of patients…