For people with cystic fibrosis (CF), the decision of whether or not to become a parent is fraught with psychological challenges, ranging from difficult fertility treatments to deep concerns about long-term health and family stigma, a new review paper highlights. The findings underscore the critical importance of providing comprehensive…
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Immune abnormalities linked to lung inflammation and damage in people with cystic fibrosis (CF) may begin early in life and persist despite treatment, a study in Australia suggested. The study, led by researchers at the Murdoch Children’s Research Institute (MCRI) and the Peter MacCallum Cancer Centre, found widespread abnormalities…
Immune cells that patrol the lungs, called alveolar macrophages, may help sustain persistent inflammation in cystic fibrosis (CF), according to a new study. Researchers found that alveolar macrophages in people with CF differed from healthy cells, showing inflammatory activity, abnormal cholesterol handling, and altered communication with other immune cells…
People with cystic fibrosis (CF) treated with CFTR modulators have a significantly lower risk of death than untreated patients, with an overall 66% lesser risk over eight years, according to a large U.S. registry study. The survival benefit was similar in magnitude in males and females. “Our study is…
Data from continuous glucose monitoring (CGM) devices, which track blood sugar levels in real time, can reveal early signs of diabetes in people with cystic fibrosis (CF), even among those who test negative for diabetes using standard tools. That’s according to a study using machine learning, a type of…
ETD001, an inhaled therapy for cystic fibrosis (CF), appeared to work better than a placebo at improving lung function in CF patients not taking CFTR modulators — approved therapies that only work in people with certain CF-causing gene mutations — in a small clinical trial. That’s according to…
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…
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…
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