News

Sevion Therapeutics and Eloxx Pharmaceuticals announced that a first healthy subject has been dosed in a Phase 1b clinical trial assessing the safety, tolerability and drug properties of ELX-02 as a potential treatment of several genetic diseases caused by nonsense mutations, including cystic fibrosis (CF). Nonsense mutations are…

Michelle Laver remembers putting a rattle in her infant daughter’s hand and closing her little fingers around it. Then she watched the rattle fall. Kate couldn’t hold on to it. Michelle and Richard Laver, founders of Kate Farms, with Kate, 11. (Photo courtesy of Kate Farms) Kate eventually…

AIT Therapeutics’ inhaled nitric oxide formulation improved the breathing and physical functioning of cystic fibrosis patients with a lung infection, a Phase 2 clinical trial shows. The preliminary results applied to the formulation’s use against nontuberculous mycobacteria, which are collectively known as the mycobacterium abscessus complex. AIT said the NO-NTM…

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…

A recent study found improvements in lung function and body mass index (BMI) among children and young adults with cystic fibrosis (CF) both in the United States and Canada from 1990 to 2013, though the American patients improved faster than those in Canada. The study, “Comparison of Nutrition and…

Researchers at the University of Alabama at Birmingham (UAB) and the University of North Carolina at Chapel Hill (UNC) have developed a new method that can quickly and easily predict which treatment is more suited for each cystic fibrosis (CF) patient. Their study, “Nasospheroids permit…

Galapagos has initiated a Phase 1 clinical trial in healthy volunteers to evaluate a new C2 corrector, called GLPG3221, developed for the treatment of cystic fibrosis (CF). The Phase 1 study, being conducted in Belgium, will evaluate the safety and tolerability of GLPG3221, and seeks to understand how the…

Cystic fibrosis (CF) patients have more bacteria but lower bacterial diversity in their lungs than people without CF — yet bacterial diversity diminishs with age in CF patients, a large multicenter study found. The study, “Airway microbiota across age and disease spectrum in cystic fibrosis,” appeared in…

Adding  investigational CFTR corrector GLPG2222 safely improved disease parameters in cystic fibrosis (CF) patients treated with Kalydeco (ivacaftor), reported the therapy’s Belgian developer, Galapagos, citing Phase 2 clinical trial data. The benefit of adding GLPG2222 was similar to that seen in an earlier study of a tezacaftor-Kalydeco combination, researchers said.

Cystic fibrosis care has seen such rapid advances that the average CF patient has likely seen a dramatic evolution in treatment strategies in their lifetime. Here are some of the biggest milestones that shaped modern-day CF treatments. (Dates are when these therapies and ideas were adopted. Many are no longer…