News

The European Medicines Agency (EMA) granted orphan drug status to AR-501, a treatment candidate for lung infections in people with cystic fibrosis (CF). AR-501, developed by Aridis Pharmaceuticals and also known as Panaecin, is an inhaled formulation of gallium citrate intended as a weekly and self-administered therapy delivered…

Vertex Pharmaceuticals is asking the U.S. Food and Drug Administration to approve a first triple combination therapy —  elexacaftor (VX-445) plus tezacaftor, and ivacaftor (Kalydeco) — to treat cystic fibrosis (CF) patients who cannot use its other disease-modifying treatments or don’t benefit as intended. Triple combinations include…

Treatment with Kalydeco (ivacaftor) improved lung function, exercise capacity, and sweat chloride concentration in cystic fibrosis (CF) patients with severe lung disease, a small retrospective analysis shows. The study, “Effectiveness of ivacaftor in severe cystic fibrosis patients and non‐G551D gating mutations,” was published in the journal Pediatric Pulmonology. Mutations…

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…

Scientists have created a miniature human pancreas-on-a-chip that may help identify the cause of cystic fibrosis-related diabetes (CFRD), a common and life-threatening complication of cystic fibrosis (CF). Researchers believe the pancreas-on-a-chip will provide insightful information about the biology of CFRD, offering a reliable way to monitor pancreatic cell responses and…

Poorer lung function in pregnant women with cystic fibrosis (CF) is associated with a lower birth weight for offspring, and a higher likelihood of cesarean section — but not with changes in lung or metabolic parameters, new research suggests. The study, titled “Pregnancy outcome in women…

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,…

Treating infants with cystic fibrosis (CF) with the antiviral therapy Synagis (palivizumab) does not improve long-term outcomes in lung health, real-world data show. Routine use of Synagis is not recommended by the American Academy of Pediatrics for babies with CF, although its use can be considered in those at…

Approved and investigative cystic fibrosis (CF) treatments known as CFTR potentiators work by binding to the same flexible protein spot — a place that acts like a hinge — and keeping it open, according to a study whose findings may lead to more effective therapies. The research, “Structural…

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,…