Researchers have identified a new class of CFTR correctors to treat cystic fibrosis (CF) that enhance the potency of currently available CFTR modulator therapies, a study shows. Data reveal that these compounds demonstrated high potency in airway cells derived from CF patients. The study, “Novel tricyclic…
News
A single dose of 4D Molecular Therapeutics’ 4D-710, an inhaled gene therapy candidate for adults with cystic fibrosis (CF), improved or stabilized lung function in three patients in a Phase 1/2 clinical trial. Nine to 12 months of follow-up data showed it led to meaningful gains in quality…
The personality traits of people with cystic fibrosis (CF) might influence their health-related quality of life (HRQoL), according to a recent study. Scientists found that patients could be clustered into two distinct personality groups and that self-reported quality of life differed significantly between them. The findings, published in “…
People with cystic fibrosis (CF) who frequently use healthcare resources accounted for almost one-half of total healthcare costs, according to a new Canadian study. Becoming a frequent high-cost CF healthcare user was linked to severe lung impairment, lung transplant, and liver cirrhosis (a chronic liver disease) with high blood…
Changes in two human voice parameters may help identify poor control of blood sugar levels in people with cystic fibrosis (CF) who have CF-related diabetes (CFRD), a pilot study reported. Lower measures on these parameters, known as fundamental frequency variation and noise-to-harmonic ratio, were linked with higher glucose,…
For people with cystic fibrosis (CF), key barriers to getting specialty care include concerns about addiction and affordability, according to a new survey of clinicians. In fact, more than one-third of respondents cited difficulties for CF patients in accessing specialists and issues with insurance and treatment costs. “In this…
For people with cystic fibrosis (CF) taking Pulmozyme (dornase alfa), adding treatment with hypertonic saline — or HS for short — did not significantly improve lung function or reduce the risk of exacerbations compared with the approved therapy alone, a new study indicates. The findings showed that…
Kaftrio (elexacaftor, tezacaftor, and ivacaftor) safely and effectively eases structural lung damage and improves lung function and the quality of life for people with cystic fibrosis (CF) who have advanced lung disease. That’s according to data from a compassionate use program in the Netherlands that let patients with severely…
Men with cystic fibrosis (CF) saw greater reductions in lung symptom exacerbations after starting treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) than their female counterparts, according to a recent study. Changes in nutritional status, lung infections, and lung function were similar between the sexes. The findings overall suggest that high-efficacy therapies…
Among children with cystic fibrosis (CF), lung function was seen to worsen faster following a bacterial infection with Pseudomonas aeruginosa, especially if the infection became chronic or ongoing, a new study reports. The link between the bacterium — commonly known as Pseudomonas or P. aeruginosa — and a…
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