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2011-06-09 07:01:00

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. The trial lasted three weeks. Participants took VX-809 for two weeks,...

2010-11-18 14:01:24

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, according to findings published Nov. 18, 2010, in the New England Journal of Medicine. The investigational drug, VX-770, appeared to improve function of what is known as CFTR--the faulty protein responsible for CF. It is among the first compounds being developed for CF that specifically targets the root cause of cystic fibrosis. Patients...

2010-11-15 12:12:44

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. An imbalance of salt and water in patients with cystic fibrosis makes their lungs clog up with sticky mucus that is prone to infection. The cause of the offending imbalance is a well-known genetic error, one that blocks the molecular expressway for...

2010-10-12 18:18:07

Study recognized for significance and importance in the world's most common genetic disease A University of Missouri researcher believes his latest work moves scientists closer to a cure for cystic fibrosis, one of the world's most common fatal genetic diseases. The Journal of Biological Chemistry has published findings by Tzyh-Chang Hwang, a professor in the School of Medicine's Department of Medical Pharmacology and Physiology and the Dalton Cardiovascular Research Center. The publication...

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2010-09-20 07:52:26

Crosstalk between ion channels points to new therapeutic strategy, Penn study finds The lab of Kevin Foskett, PhD, the Isaac Ott Professor of Physiology at the University of Pennsylvania School of Medicine, has found a possible new target for fighting cystic fibrosis (CF) that could compensate for the lack of a functioning ion channel in affected CF-related cells. Their finding appears in the Journal of Clinical Investigation. The team explored the role of CFTR, the chloride ion channel...

2008-10-21 09:00:53

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. VX-770, an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, was well-tolerated when dosed orally as 150mg or 250mg twice daily for 28 days. In this analysis, no patients discontinued...

2008-07-31 03:00:20

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). The gene which harbors mutations responsible for disease was identified in 1989, after which the protein it encodes was determined to function as a chioride channel that indirectly controls...

2008-06-26 06:02:10

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. It is the most common inherited condition in white populations, and is increasingly recognised in non-white populations. CF is an autosomal recessive disorder caused by a mutation at the CF transmembrane conductance regulator gene that affects various cellular activities. These include transport of...


Latest Sweat test Reference Libraries

Cystic Fibrosis
2013-07-19 15:03:45

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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Word of the Day
malpais
  • The ragged surface of a lava-flow.
'Malpais' translates from Spanish as 'bad land.'