Lumacaftor (VX-809) — one of the medications in the CFTR modulator Orkambi — led to a significant protein reorganization in two cellular compartments in a cell model of cystic fibrosis (CF), causing them to more closely resemble healthy cells, a study showed. The findings expand the potential mechanisms…
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Cincinnati Children’s Hospital Medical Center will use Epistemic AI’s platform, a state-of-the-art artificial intelligence (AI) research tool for the life sciences, to augment its research into cystic fibrosis (CF) and other lung conditions. The Boomer Esiason Foundation (BEF) venture philanthropy initiative helped bring this capability to scientists at Cincinnati…
Gut bacteria entering the lungs during acid reflux episodes may promote lung inflammation and poorer lung function in cystic fibrosis (CF) patients, a new study reports. These findings may explain why people with CF and acid reflux tend to have poorer lung function. The study, “Exposure to bile…
A type of ring-shaped molecule called a macrocycle can help CFTR — the faulty protein in cystic fibrosis (CF) — go to the cell’s membrane, where it exerts its action, and work better in lab-grown cells carrying the most common CF-causing mutation, a study showed. These benefits were associated…
Treatment with Kalydeco (ivacaftor) significantly reduces the levels of inflammatory molecules in the lungs of preschool-age children with cystic fibrosis (CF), according to a small study. A reduction in lung inflammation was not observed in preschoolers with CF who were treated with Orkambi (lumacaftor/ivacaftor). These findings add…
Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…
Destiny Pharma has received an award from the Cystic Fibrosis Foundation (CFF) to evaluate its investigational compound, XF-73, as a potential treatment for methicillin-resistant Staphylococcus aureus (MRSA) lung infections in people with cystic fibrosis (CF). The study will be carried out by researchers affiliated with the CFF,…
Children with cystic fibrosis (CF) who have lung infections due to CF-related bacteria, especially Pseudomonas aeruginosa, were found to have higher levels in their bloodstream of biomarkers of oxidative stress and related tissue damage. These high levels were accompanied by reduced lung function and worse nutritional status, according…
The government of Ontario has expanded coverage of Vertex Pharmaceuticals’ triple-combination treatment Trikafta for children, 6 and older, with cystic fibrosis (CF) and at least one copy of the most common CF-causing mutation, called F508del. The announcement comes a few months after Health Canada expanded Trikafta’s previous label,…
A technique called 2D shear wave elastography, which measures the stiffness of tissues, could be useful for detecting liver disease in people with cystic fibrosis. That’s according to the study, “2D Shear Wave Elastography, a promising screening tool for Cystic Fibrosis liver disease, shows a correlation…
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