Margarida Maia, PhD, science writer —

Gene therapy was found to work as well as a combination of the three CFTR modulators in Trikafta to restore the function to the CFTR protein, which is faulty in cystic fibrosis (CF), with the potential to work for all patients, regardless of their disease-causing mutation. That’s according…

While physical health and life quality gains can be evident in people using CFTR modulators, a group of disease-modifying therapies for cystic fibrosis (CF), their mental well-being needs more attention, according to the Wellness in the Modulator Era (Well-ME) study. Nearly 1 in every 4 patients reported poorer…

The first healthy volunteers have been dosed in Australia as part of two separate Phase 1 clinical trials of SION-719 and SION-451 — two second-generation NBD1 stabilizers that Sionna Therapeutics is developing to restore function to the CFTR protein, which is faulty in cystic fibrosis (CF). The goal…

Mice mimicking cystic fibrosis (CF) have trouble forming and taking up fat droplets, which limits normal growth and optimal nutrition, a study found, suggesting that a high-fat diet may not be helping patients as much as thought and needs to be reconsidered. According to the researchers, a key finding…

Sionna Therapeutics has gained the rights to develop and market three of AbbVie’s clinical-stage compounds, a move that expands the company’s pipeline of small molecules that are designed to restore the function of the CFTR protein, which is faulty or absent in cystic fibrosis (CF). The compounds…

Despite treatment with Kalydeco (ivacaftor), females with cystic fibrosis (CF) continue to experience more lung exacerbations — periods when CF respiratory symptoms suddenly worsen — than do males, for reasons that are not well understood, a large patient registry study reported. “Our findings demonstrate that sex disparities…

Despite recent guidelines recommending nutrient consumption similar to the general population, many adults and late adolescents with cystic fibrosis (CF) still follow a high-fat, high-energy diet, according to a review done by researchers in Australia. The findings suggest that “overall, diet intakes of adults with CF appear to be…

Tezacaftor, a medication used to treat cystic fibrosis (CF), may unexpectedly trap certain fats in the lungs, diverting their use as chemical raw material that the body converts into ceramides, which are needed for the brain to develop and respond to stress, according to…

Children with cystic fibrosis (CF) who are being treated with Symdeko (ivacaftor/tezacaftor) or Orkambi (ivacaftor/lumacaftor) experience few to no symptoms of chronic rhinosinusitis and maintain a normal sense of smell, a study in Denmark found. Although small, the study suggests that not only Trikafta, a triple…

CFTR modulators may help control glucose (sugar) levels in the blood of people with cystic fibrosis (CF), according to researchers in Israel, who called for regular oral glucose tolerance testing to adjust insulin dosing in diabetes and keep glucose from dropping too low. The study, “Long-Term therapy with…