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Latest nonsense mutation cystic fibrosis Stories

2014-06-30 08:30:32

SOUTH PLAINFIELD, N.J., June 30, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the initiation of a global confirmatory Phase 3 clinical trial of Translarna(TM) (ataluren), an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). Nonsense mutations within cystic fibrosis are categorized as Class I mutations, a severe form of CF that results in little or no production of the CFTR protein. The Phase 3 confirmatory trial is...

2011-07-01 07:00:00

SOUTH PLAINFIELD, N.J., July 1, 2011 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the publication of data from a Phase 2 study of ataluren, an investigational new drug, in adults with nonsense mutation cystic fibrosis (nmCF) in the European Respiratory Journal. The published three-month data showed that treatment with ataluren resulted in statistically significant improvements in chloride channel activity, CF-related cough and positive trends in lung function. (Logo:...

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,...

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
maffling
  • To stammer.
  • Present participle of maffle, to stammer.
  • A simpleton.
The word 'maffle' may come from a Dutch word meaning 'to move the jaws' or a French word meaning 'having large cheeks'.