Latest ΔF508 Stories
The study, led by Martina Gentzsch, PhD, provides evidence that could help drug developers improve compounds aimed at correcting CFTR proteins in cystic fibrosis patients.
With over 1,900 mutations in the genes responsible for cystic fibrosis (CF), separating the harmful alterations from the benign ones has been a long, arduous undertaking for geneticists. Until now, that is.
Scientists at the Hospital for Sick Children in Toronto have established that a drug recently approved by the U.S. Food and Drug Administration to treat a rare form of cystic fibrosis works in an unconventional way.
By developing software that uses 3-D models of proteins involved in cystic fibrosis, a team of scientists at Duke University has identified several new molecules that may ease the symptoms of the disease.
A recent study led by Gergely Lukacs, a professor at McGill University's Faculty of Medicine, Department of Physiology, and published in the January issue of Cell, has shown that restoring normal function to the mutant gene product responsible for cystic fibrosis (CF) requires correcting two distinct structural defects.
Researchers have identified an unconventional path that may correct the defect underlying cystic fibrosis.
BETHESDA, Md., June 9, 2011 /PRNewswire-USNewswire/ -- Vertex Pharmaceuticals Incorporated and the Cystic Fibrosis Foundation today announced promising results from an ongoing Phase 2 study evaluating combinations of VX-770 and VX-809, potential medicines designed to treat the defective protein that causes cystic fibrosis. The study enrolled 62 people with two copies of the most common CF mutation, known as Delta F508.
Patients on drug had significant weight gain, reduced sweat chloride and were less likely to experience a pulmonary exacerbation BETHESDA, Md., Feb.
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