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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.
An investigational drug targeting a defective protein that causes cystic fibrosis has been shown to improve lung function in a small study of CF patients.
Researchers at the University of North Carolina at Chapel Hill School of Medicine have demonstrated that the gene mutated in cystic fibrosis not only controls traffic on the chloride highway, but also keeps the sodium highway from being overused.
A University of Missouri researcher believes his latest work moves scientists closer to a cure for cystic fibrosis, one of the worldâ€™s most common fatal genetic diseases.
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Vertex Pharmaceuticals, a global biotechnology company, has announced positive results from a preliminary analysis of data from part two of the Phase IIa clinical trial of the investigational oral drug VX-770 in cystic fibrosis patients who carry the G551D CFTR mutation.
By Kleven, Daniel T McCudden, Christopher R; Willis, Monte S Cystic fibrosis (CF) is the most common lethal genetic disease in Caucasians, occurring in 1/2,500 births.12 In its most common form, CF manifests as progressive lung dysfunction, pancreatic insufficiency, and intestinal disease (see Figure 1).
By Knott, Laurence THE BASICS Proactive management can improve patient survival. By Dr Laurence Knott Cystic fibrosis (CF) affects one in 2,500 newborns and has a UK prevalence of 8,000.
Cystic fibrosis, also called mucoviscidosis, is an autosomal recessive genetic disorder of the viscous secretions in the body. In turn, it effects the lungs, pancreas, liver, and intestines, as well as all other exocrine glands in the body. The most common genetic mutation that causes CF is a deletion of three nucleotides that results in a loss of phenylalanine, an amino acid at the 508th position on the protein. It should be noted, however, that there are over a thousand other mutations that...
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