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"South Plainfield, N.J., April 23, 2007 — New preclinical data published online in the current edition of the journal Nature show that PTC124, an investigational new drug designed to bypass nonsense mutations, was efficacious in a preclinical model of Duchenne muscular dystrophy (DMD). It is estimated that approximately 13 percent of the cases of DMD are due to nonsense mutations. PTC Therapeutics, Inc., which discovered and is developing PTC124, has catalogued over 1,800 distinct genetic disorders where nonsense mutations are the cause of the disease in a significant percentage of patients. Nonsense mutations inactivate gene function and are known to cause anywhere from five to 70 percent of the individual cases of most inherited diseases, such as cystic fibrosis (10%) and Hurler’s syndrome (70%)"
