A Phase 1 clinical trial of an experimental inhalation gene therapy for cystic fibrosis (CF) — KB407 by Krystal Biotech — was given clearance to start by the U.S. Food and Drug Administration (FDA). Expected to begin this year, the study aims to enroll 20 adults with CF who…
Adults with cystic fibrosis (CF) are willing to accept reductions in lung function and life expectancy as a trade-off for a reduced treatment burden, a survey of patient preferences suggested. Over all other outcomes, the participants preferred a treatment choice that would extend their life expectancy by 10 years…
Allergies to beta-lactam antibiotics were the most commonly confirmed antibiotic allergy in children with cystic fibrosis, according to a small study in Turkey. Sensitivity to multiple types of antibiotics occurred in one-third of children with an allergy, data showed. Having an immediate reaction to a medication and an allergy…
Mothers who care for children with cystic fibrosis (CF) often experience depression, burnout, caregiver burden, and negative attitudes toward their children, a study suggests. These caregiving struggles may increase with more hospital visits and hospitalizations, and having a lower family income, more children, and another child with a chronic…
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…
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