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Latest Ataluren Stories

2013-06-26 12:53:53

A drug developed to treat genetic diseases such as Duchenne muscular dystrophy and cystic fibrosis may need a radical rethink. In a new study published on 25 June in the open access journal PLOS Biology, researchers question the mechanistic basis of the drug called PTC124 (also known as Ataluren), casting doubt as to whether it has the molecular effects that are claimed for it. This may have implications for its effectiveness in treating genetic diseases. An estimated 10% of all human...

2012-12-04 15:00:32

Biologists at Mainz University achieve repair and read-through of stop mutations responsible for the disease After years of basic research, scientists at Johannes Gutenberg University Mainz (JGU) are increasingly able to understand the mechanisms underlying the human Usher syndrome and are coming ever closer to finding a successful treatment approach. The scientists in the Usher research group of Professor Dr. Uwe Wolfrum are evaluating two different strategies. These involve either the...

2010-12-21 07:00:00

SOUTH PLAINFIELD, N.J., Dec. 21, 2010 /PRNewswire/ -- PTC Therapeutics, Inc. announced today that it has completed enrollment of a Phase 3 clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). (Logo: http://photos.prnewswire.com/prnh/20010919/PTCLOGO ) The 48-week study is designed to determine whether ataluren can improve lung function in patients with nmCF. The trial has enrolled 238 patients at 36 sites in North America,...


Word of the Day
Cthulhu
  • A gigantic fictional humanoid alien god being described with a head resembling an octopus and dragon wings and claws, around whom an insane cult developed.
  • Pertaining to the mythos of Cthulhu and additional otherworldly beings created by H. P. Lovecraft or inspired by his writings and imitators.
This word was invented in 1926 by H.P. Lovecraft for his short story, 'The Call of Cthulhu.' 'Cthulhu' may be based on the word 'chthonic,' which in Greek mythology refers to the underworld.
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