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A potential gene-specific therapy for cystic fibrosis (CF) may lie in antisense oligonucleotides, or ASOs, that enhance the production and activity of the truncated CFTR protein caused by the W1282X mutation, a cell study suggested. In combination with approved CFTR modulator therapies, candidate ASOs were seen to raise CFTR-W1282X…

Avecris and Nosis Biological Sciences have won the Cystic Fibrosis (CF) Foundation’s inaugural “Golden Ticket” Competition. The companies will each receive one year of free office and lab space at Bakar Labs and access to the CF Foundation’s network of resources to develop genetic therapies for CF.

Infusions with a hormone called GLP-1 boosted insulin production in cystic fibrosis (CF) patients with impaired glucose tolerance, according to data from a small study. The results suggest that GLP-1 agonists — medications that mimic the hormone’s activity, but last longer in the body — may be useful as…

People with cystic fibrosis (CF) who use inhaled antibiotics are at more than three times higher risk of becoming infected with the fungus Aspergillus fumigatus, according to a new study. Study results showed no association between Aspergillus infection and lung function decline, and available data demonstrated no clear benefits of treating the…

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…

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…