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Last updated on April 16, 2014 at 1:21 EDT

Latest Chloride channel Stories

2014-03-12 08:29:44

BOULDER, Colo., March 12, 2014 /PRNewswire/ -- N30 Pharmaceuticals, Inc. ("N30 Pharma") announced today that it has begun oral dosing of N91115 in a Phase 1 clinical trial. N91115, a novel inhibitor of S-nitrosoglutathione reductase (GSNOR), is being developed for the treatment of cystic fibrosis (CF). In preclinical studies, N91115 has been shown to rescue or "correct" F508del-CFTR, the most commonly occurring mutation in CF. It is the first oral GSNOR inhibitor to enter clinical...

2013-03-22 09:42:47

Data about defective regulatory protein described in Proceedings of the National Academy of Sciences A little more than a year after the FDA approved Kalydeco (Vx-770), the first drug of its kind to treat the underlying cause of cystic fibrosis, University of Missouri researchers believe they have found exactly how this drug works and how to improve its effectiveness in the future. Described in the current issue of the Proceedings of the National Academy of Sciences, MU researchers have...

2013-03-13 08:30:43

BOULDER, Colo., March 13, 2013 /PRNewswire/ -- N30 Pharmaceuticals, Inc. ("N30 Pharma") a leader in the development of therapeutics that target S-nitrosoglutathione reductase (GSNOR), announced today that it has administered the first dose in its Phase 1b/2a clinical trial of N6022 in patients with cystic fibrosis (CF). The study is a multicenter, double-blind, randomized, placebo-controlled evaluation of ascending doses of N6022 in patients who have two copies of the F508del-CFTR mutation,...

2012-03-12 14:31:32

New research could provide roadmap for more effective drug discovery for CF 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. This finding could point to more effective therapeutic strategies for CF in the future. CF, a...

2011-09-19 21:57:12

Cystic fibrosis (CF), a chronic disease that clogs the lungs and leads to life-threatening lung infections, is caused by a genetic defect in a chloride channel called cystic fibrosis transmembrane conductase regulator (CFTR). Although scientists do not fully understand how or why this defect occurs, a team of researchers at The Hospital for Sick Children (SickKids) in Toronto, Ontario, Canada has found a promising clue: a protein called ubiquitin ligase Nedd4L. In a study led by Daniela...

2011-09-19 21:56:15

When researchers discovered the primary genetic defect that causes cystic fibrosis (CF) back in 1989, they opened up a new realm of research into treatment and a cure for the disease. Since then, scientists have been able to clone the defective gene and study its effects in animals. Now researchers at the University of North Carolina at Chapel Hill have developed a technique for observing the defects at work in human tissue donated by patients with CF. This technique has yielded an...

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...

2010-11-15 07:00:00

SOUTH PLAINFIELD, N.J., Nov. 15, 2010 /PRNewswire/ -- PTC Therapeutics, Inc. today announced the publication of data from a Phase 2a clinical trial of ataluren in children with nonsense mutation cystic fibrosis (nmCF) in the American Journal of Respiratory and Critical Care Medicine. The published data show that treatment with ataluren, an investigational new drug, resulted in statistically significant improvements in the production and function of cystic fibrosis transmembrane...

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...