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Latest PTC124 Stories

2011-07-01 13:32:52

New treatment approach shall soon be ready for use in Usher syndrome patients Usher syndrome is the most common form of combined congenital deaf-blindness in humans and affects 1 in 6,000 of the population. It is a recessive inherited disease that is both clinically and genetically heterogeneous. In the most severe cases, patients are born deaf and begin to suffer from a degeneration of the retina in puberty, ultimately resulting in complete blindness. These patients experience major...

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:...

2010-12-23 00:00:54

The new Renilla-Gloâ“ž¢ Luciferase Assay brings the convenience of the "add, mix, measure" assay format to applications using the sea pansy (Renilla reniformis) luciferase. Madison, WI (PRWEB) December 22, 2010 The new Renilla-Gloâ“ž¢ Luciferase Assay brings the convenience of the "add, mix, measure" assay format to applications using the sea pansy (Renilla reniformis) luciferase. The assay is ideal for screening in applications...

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...

2010-01-19 08:00:00

SOUTH PLAINFIELD, N.J., Jan. 19 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently. This trial is evaluating the best methods for measuring functional abilities in patients who have lost independent mobility. Patients with nmDBMD develop...

2009-12-08 07:00:00

SOUTH PLAINFIELD, N.J., Dec. 8 /PRNewswire/ -- Data published online in the medical journal Muscle and Nerve support the use of the six-minute walk test (6MWT) as an outcome measure in PTC Therapeutics, Inc.'s ongoing registration-directed clinical trial of ataluren in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDMD/BMD). The study results, which will also be published in an upcoming print issue of the journal, characterize the limitations on walking by patients...

2009-10-26 07:00:00

SOUTH PLAINFIELD, N.J., Oct. 26 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced it is expanding the development of ataluren, an investigational new drug, to a third indication with the initiation of a Phase 2a clinical trial in nonsense mutation hemophilia A (nmHA) and hemophilia B (nmHB). Hemophilia is a rare and debilitating genetic disorder that causes loss of blot clotting proteins and can lead to serious, recurrent bleeding episodes. It is estimated that nonsense...

2009-09-10 07:00:00

SOUTH PLAINFIELD, N.J., Sept. 10 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the initiation of a Phase 3 trial of ataluren (formerly PTC124(R)), an investigational protein restoration therapy in patients with nonsense mutation cystic fibrosis (nmCF). Patients with CF lack adequate levels of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a chloride channel necessary for normal function of the lung, pancreas, liver, and other organs. In nmCF, an...


Word of the Day
drawcansir
  • A blustering, bullying fellow; a pot-valiant braggart; a bully.
This word is named for Draw-Can-Sir, a character in George Villiers' 17th century play The Rehearsal.
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