Blackstone Life Sciences is investing $250 million in Anagram Therapeutics to advance ANG003, an oral enzyme replacement therapy (ERT) for exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). ANG003 is designed to reduce the “enormous, disruptive” pill burden for CF patients with EPI, which can amount…
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An early clinical trial testing inhaled cystic fibrosis (CF) treatment VX-522 — being developed for adults with CF who are not eligible for certain available therapies — has been terminated before its completion due to “persistent tolerability issues,” according to an update from Vertex Pharmaceuticals. Vertex, which was…
Having a family history of diabetes may significantly increase the risk of developing cystic fibrosis-related diabetes (CFRD), a common complication of cystic fibrosis (CF), according to a new U.S. study. The researchers found that adults with CF who had a family history of diabetes, particularly type 2 diabetes,…
Children with cystic fibrosis who carry two Trikafta-responsive mutations are significantly more likely to reach normal or near-normal sweat chloride levels, suggesting stronger restoration of CFTR protein function than children with one responsive and one nonresponsive mutation, according to a real-world study. While this biochemical benefit ultimately did…
Trikafta (elexacaftor/tezacaftor/ivacaftor) is generally effective at easing sinus issues in people with cystic fibrosis (CF) who have already undergone a lung transplant, but more research is needed to evaluate whether it can improve nutritional status or lung function in transplant recipients, according to a new analysis. Researchers also…
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…
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…
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