Advocates around the globe are gearing up for a variety of events and digital campaigns this week to kick off Cystic Fibrosis Awareness Month. From neon-lit bike rides in California to national walks across Canada, these May initiatives seek to fund life-saving research and…
News
A small clinical trial testing Sionna Therapeutics‘ SION-719 as an add-on treatment for cystic fibrosis (CF) — to be used alongside the approved CF therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) — has finished enrollment. The developer announced the milestone in a company press release, noting that top-line data from the…
People with cystic fibrosis (CF) taking Trikafta usually report notable improvements in their quality of life — even if they don’t show substantial gains in objective measures of lung function — soon after starting treatment with the approved oral medication. That’s according to a new real-world study, which…
The use of combined hormonal contraceptives by women with cystic fibrosis (CF) who are not taking CFTR modulators increases by 53% the risk of developing CF-related diabetes (CFRD), a study in the U.S. found. Conversely, combined hormonal contraceptive “use was not associated with increased CFRD incidence among modulator…
Respiratory symptoms are key drivers for starting Kaftrio (elexacaftor/tezacaftor/ivacaftor) among adults with cystic fibrosis (CF) who have stable, near-normal breathing ability, known as preserved lung function, a survey study in Europe has found. In surveying CF centers across the continent, the research team found that clinicians were more likely…
A therapy created using a miniaturized antibody can correct the functionality of the defective CFTR protein in people with cystic fibrosis (CF) caused by the F508del mutation. Preclinical data indicate that this nanobody can act in synergy with Trikafta (ivacaftor/tezacaftor/elexacaftor), an approved treatment for CF. In combination,…
Gene therapy using circular forms of DNA called plasmids may help treat lung disease in cystic fibrosis (CF), based on early lab studies, regardless of the disease-causing gene mutation, a study suggests. Optimized plasmids boosted production of the fully mature CFTR protein, which is missing or not working properly…
Trikafta was associated with significant reductions in healthcare use, including hospitalizations related to cystic fibrosis (CF) complications affecting organs and systems outside the lungs, according to an insurance claims analysis. “Results of this study suggest a multisystemic benefit of [Trikafta] therapy with reductions in extrapulmonary…
In people with cystic fibrosis (CF), the timing of organ transplant — lung, liver, or both — relative to infection with the SARS-CoV-2 virus that causes COVID-19 did not affect the risk of severe outcomes, according to a large international study. Patients with poorer lung…
Among people with cystic fibrosis (CF), certain measures of blood vessel health — especially regarding the aorta, the body’s main artery — tend to vary according to biological sex, a new study reports. Researchers think these variations may help explain sex-specific differences in survival outcomes among people with CF,…
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