News

A test of physical function called Glittre-ADL may be better than the standard six-minute walk test at identifying physical limitations in people with cystic fibrosis (CF), a study reports. “To our knowledge, this is the first study to evaluate the performance of CF patients on TGlittre and to compare…

In a recent study, scientists described the process through which they discovered GLPG2737, an investigational CFTR modulator considered by Galapagos and AbbVie to be a possible treatment for cystic fibrosis (CF). The molecule was found in early work to have an additive effect when combined with other types…

Treatment with Trikafta (ivacaftor/tezacaftor/elexacaftor) eases nose and sinus symptoms in eligible patients with cystic fibrosis (CF), and the improvement in nasal symptoms is accompanied by an increase in nasal nitric oxide (nNO), a biomarker. The findings suggest “nNO could be used as an objective, noninvasive outcome measure for…

A five-year study led by scientists at Emory University in Georgia aims to identify new ways to improve quality of life for people with cystic fibrosis (CF). The study, which will be led by Dio Kavalieratos, PhD, director of research at the Emory Palliative Care Center, is funded by…

A novel treatment strategy for cystic fibrosis (CF) that’s based on a cellular structure called the porosome showed promise in cell and animal models of CF caused by the common F508del mutation. In the cell model, this approach was more potent than tezacaftor and ivacaftor, which are CFTR…

Highly effective CFTR modulators, which target the cause of cystic fibrosis (CF), may ease signs of low iron levels in some patients with the disease, but low iron levels may remain even with treatment with the medications, a study finds. The scientists also emphasized that people with CF who…

Biopharma and biotech companies have launched more than 450 clinical trials into potential new treatments for cystic fibrosis (CF) since 2018, according to a report from Novotech, a contract research organization that specializes in helping drug developers to run trials. “There are ample opportunities presented by advancements in…

The CFTR modulator therapy Kalydeco (ivacaftor) can safely be given to babies with cystic fibrosis (CF) as young as 4 weeks old, according to data from a Phase 3 clinical trial. The findings were the basis of the U.S. Food and Drug Administration (FDA)’s decision to expand…

Treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) led to gains in body mass among cystic fibrosis (CF) patients that were largely driven by increases in various types of fat and low-density muscles, a study found. High-density muscle, which is stronger than its lower-density counterparts, didn’t change with the treatment, sold as…

While Kaftrio — sold as Trikafta in North America — can improve lung function in children with cystic fibrosis (CF), treatment effects are unlikely to be as dramatic in real-world practice as those reported in clinical trials. Rather, for eligible children without substantial lung problems, Kaftrio’s use can…