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PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy
Date:10/11/2007

a Wong, M.D., Associate Professor of Pediatrics and Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, who presented the data today at CNS and is one of the trial's lead investigators. "Based on the findings from this study, we believe that the safety profile of PTC124 supports continued testing in longer-term studies."

"These results add to the growing body of safety data for PTC124, which has now been evaluated in more than 150 subjects, including patients with both DMD and cystic fibrosis. The safety profile has consistently shown that PTC124 appears well tolerated," said Langdon Miller, M.D., Chief Medical Officer of PTC. "We are looking forward to presenting additional activity data from this study next week at the World Muscle Society meeting in Italy."

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children). More information regarding DMD is available through the Muscular Dystrophy Association (http://www.mdausa.org) and the Parent Project Muscular Dystrophy (http://www.parentprojectmd.org).

About PTC124

PTC124 is an orally delivered investigational new drug in Phase 2 clinical development for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely halt the translation process, producing a shortened, non- functional protein. PTC124 has restored production of full-length, functional proteins in preclinical genetic disease models harboring nonsense mutations. In Ph
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SOURCE PTC Therapeutics, Inc.
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