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Latest ΔF508 Stories

Cystic Fibrosis Genetics Breakthrough
2013-08-26 12:46:54

Brett Smith for redOrbit.com - Your Universe Online With over 1,900 mutations in the genes responsible for cystic fibrosis (CF), separating the harmful alterations form the benign ones has been a long, arduous undertaking for geneticists. In a new report published in Nature Genetics, researchers describe major progress in determining which mutations are which, increasing the number of recognized CF-causing mutations from 22 to 127. The new findings represent 95 percent of the...

2012-10-29 12:07:01

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. Their results reveal new possibilities for treating various forms of cystic fibrosis. Cystic fibrosis is an inherited disease afflicting about 70,000 people around the world. Cystic fibrosis patients carry a defective gene that disables or destroys its protein product, which...

2012-04-20 10:14:03

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. Computer algorithms created by the team predict how well a given molecular structure will block a basic protein-protein interaction known to occur in cystic fibrosis. To test the predictions, the scientists synthesized the molecules and measured how well they attached to one of the...

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...

2011-09-01 16:42:25

Researchers have identified an unconventional path that may correct the defect underlying cystic fibrosis, according to a report in the September 2nd issue of the journal Cell, a Cell Press publication. This new treatment dramatically extends the lives of mice carrying the disease-associated mutation. Cystic Fibrosis is caused by a mutation in a gene responsible for the transport of ions across cell membranes. This gene encodes a protein channel, called the cystic fibrosis transmembrane...

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,...

2011-02-23 07:01:00

Patients on drug had significant weight gain, reduced sweat chloride and were less likely to experience a pulmonary exacerbation BETHESDA, Md., Feb. 23, 2011 /PRNewswire-USNewswire/ -- The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis, showed promising results in a Phase 3 clinical trial. The trial was designed to evaluate patients age 12 and up who carry...

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...


Word of the Day
call-note
  • The call or cry of a bird or other animal to its mate or its young.
'Call-note' is newer than 'bird-call,' which originally referred to 'an instrument for imitating the note of birds' but now also refers to 'the song or cry of a bird.'
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