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PTC Therapeutics Announces Additional Positive Interim Phase 2,Results of PTC124 in Duchenne Muscular Dystrophy

nd teachers reported that boys participating in the study had improvements in terms of greater activity level and increased endurance during treatment. Individual subjects at both dose levels demonstrated some improvements in upper and lower muscle strength however in the overall analysis the magnitude of change was not statistically significant.

PTC124 was well tolerated among the 26 patients included in the study. Adverse events were infrequent, mild to moderate in severity, and did not result in therapy interruptions or discontinuations. There were no safety concerns based on physical examinations, vital sign measurements, electrocardiograms or laboratory parameters. Compliance with PTC124 treatment was excellent at both dose levels.

Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics, stated, "In addition to the clinical proof-of-concept data we disclosed late last year, these new insights provide us with further evidence supporting the potential of PTC124 in genetic disorders due to a nonsense mutation. The findings in the DMD trials are consistent with the results observed in Phase 2 clinical trials of PTC124 in patients with cystic fibrosis and with preclinical results in the DMD mouse model that were recently published in Nature. We are eager to extend testing of this concept into other nonsense-mediated genetic disorders."

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 (www.mdausa.org) and the Parent Pro
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