Results of three global clinical trials of a new triple-combination CFTR modulator therapy for cystic fibrosis (CF) —  vanzacaftor, tezacaftor and deutivacaftor — are expected shortly, Vertex Pharmaceuticals, the therapy’s developer, announced. Two of these Phase 3 trials, SKYLINE 103 (NCT05076149) and SKYLINE 102 (NCT05033080), which tested the…
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Sail Biomedicines’ experimental programmable RNA technology, called Endless RNA or eRNA, appears to result in long-lasting production of the protein that is missing or faulty in people with cystic fibrosis (CF), according to research funded by the CF Foundation. Producing the protein over an extended period of…
Clarametyx Biosciences has completed a $33 million financing round to support its development of CMTX-101, a non-antibiotic treatment candidate for persistent bacterial infections in people with cystic fibrosis (CF). The funding contributed to the start of a Phase 1b/2a clinical trial of CMTX-101 as an adjunct therapy to standard-of-care…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to BX004, BiomX’s inhaled phage therapy being developed for chronic Pseudomonas aeruginosa pulmonary infections in people with cystic fibrosis (CF). The designation seeks to encourage new therapies for rare diseases, those affecting fewer than 200,000…
Treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) improved the pancreatic function of two women with cystic fibrosis (CF), allowing them to stop their pancreatic enzyme replacement therapy (PERT). The women each had a combination of an F508del mutation, which leads to the abnormal folding of the CFTR protein, and…
Mental health issues — namely, attention-deficit/hyperactivity disorder (ADHD) and oppositional defiant disorder (ODD) — in children with cystic fibrosis (CF) may be associated with a poorer adherence to treatments, more hospitalizations, and higher use of antibiotics, according to a recent report. “These initial findings suggest that mental health screening…
A high burden of infection-causing bacteria persisted in the lungs of people with cystic fibrosis (CF) who were treated with Trikafta (elexacaftor/tezacaftor/ivacaftor) in a recent study, despite an increase in the overall diversity of microbes and improvements in lung function being seen. The changing dynamics of this microbial…
Various social and environmental factors in different locations across the U.S. influenced a decline in lung function among adolescents and young adults with cystic fibrosis (CF), a large-scale analysis demonstrated. CF patients with early lung function decline tended to live in areas with more social-environmental adversity, such as higher…
Treatment with the inhaled antibiotic aztreonam can stabilize lung function in people with cystic fibrosis (CF) who have chronic infections of the bacterium Pseudomonas aeruginosa. That’s according to “Inhaled aztreonam lysine in the management of Pseudomonas aeruginosa in patients with cystic fibrosis: real-life effectiveness,”…
In people with cystic fibrosis (CF), six months of treatment with Kaftrio — sold as Trikafta in the U.S. — significantly reduced the blood levels of molecules called chemokines that are involved in inflammation, new research shows. Noting that “exaggerated inflammation is a major driver of disease progression…
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