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Eloxx Pharmaceuticals’ experimental therapy ELX-02 was generally safe and resulted in a significant reduction in sweat chloride levels — a biomarker of cystic fibrosis (CF) — in patients carrying the most common CF-causing nonsense mutation, according to top-line data from a Phase 2 clinical program. While the short…

Molecules produced by the environmental bacteria Pseudomonas aeruginosa, called alkyl-quinolones derivatives — which regulate bacterial gene activity and act in cell-to-cell communication — may be useful markers for better management of patients with cystic fibrosis (CF), a study suggests. These derivatives also may enable a more clear definition…

Note: This story was updated Nov. 18, 2021, to correct information about Pulmozyme’s administration method. Bronchitol (mannitol) plus Pulmozyme (dornase alfa) is better than Pulmozyme alone to help loosen mucus in the lungs in children with cystic fibrosis (CF), and this seems to translate into better lung function. The…

A branch of the European Medicines Agency (EMA) has recommended expanding Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to include children with cystic fibrosis (CF) as young as age 6 who have at least one F508del mutation. The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP),…

People with cystic fibrosis (CF) who have mild depression or anxiety report heightened stress, with significantly lower scores on 10 of 12 measures of health-related life quality as compared with the general population of CF patients, new research shows.  According to…

Despite longer hospital stays and higher costs, adults with cystic fibrosis (CF) do not have a greater risk of death from surgery to remove the gallbladder — called a cholecystectomy — compared with people who do not have CF, a study reported. “Importantly, we found that cholecystectomy had very…

Elexacaftor (VX-445), a next-generation CFTR corrector that is part of the triple-combination therapy Trikafta, is also a potentiator, a study reports. Results further showed that in combination with ivacaftor, sold as Kalydeco and also a potentiator, elexacaftor boosts ion flow across CFTR, the…

Use of inhaled hypertonic saline can lessen cystic fibrosis (CF)-related lung damage in young children with the disease, data from a clinical trial indicates. Based on these finding, “we can conclude that hypertonic saline is an effective, safe, and cheap treatment option for preschool children with CF,” said Harm Tiddens,…

Trikafta treatment can reduce the frequency of pulmonary exacerbations — times when lung symptoms get markedly worse — for people with cystic fibrosis (CF). That’s according to an analysis of real-world data shared at this year’s North American Cystic Fibrosis Conference (NACFC), in an oral presentation, “…

Personalized treatment with bacteria-infecting viruses, called bacteriophages, could be used to combat Pseudomonas aeruginosa, the main bacteria involved in lung infections in people with cystic fibrosis (CF), both by killing the bacteria and making them less resistant to antibiotics. Bacteriophage therapy “is safe, decreases P. aeruginosa titers [levels], and…