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PTC Therapeutics Announces Additional Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy
Date:10/18/2007

le improvement in the staining for dystrophin from muscle biopsies. Response did not appear to be dependent on type of nonsense mutation.

Blood levels of muscle-derived creatine kinase were also measured as assessments of muscle integrity. Statistically significant reductions in the concentrations of muscle-derived creatine kinase were observed during PTC124 treatment. In addition, several parents and teachers reported that boys participating in the study had improvements in terms of greater activity level and increased endurance during the study duration.

"We are very encouraged by these results, which show improvements in critical biomarkers of DMD," said presenter and study investigator, Carsten Bonnemann, M.D., Assistant Professor Neurology and Pediatrics, Children's Hospital of Philadelphia and University of Pennsylvania School of Medicine and Co-Director of the Neuromuscular Program, Children's Hospital of Philadelphia. "The combined in vitro and in vivo evidence of enhanced dystrophin expression and reduced muscle fragility offer signals of pharmacological activity that we hope to translate into potential clinical benefit for patients with DMD."

"Coupled with the emerging safety profile of PTC124, these data provide the impetus for moving forward rapidly to initiate longer-term studies for boys with DMD," said Langdon Miller, M.D., Chief Medical Officer of PTC. "We are actively working with our clinical investigators and the regulatory agencies to finalize plans for additional clinical trials and we look forward to commencing these studies in the coming months."

Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics added, "These results, combined with the data presented earlier this month at the Child Neurology Society meeting and North American Cystic Fibrosis Conference, further support our belief that PTC124 represents a paradigm shift in the treatment of genetic disorders. Our future plans for PTC124 inc
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SOURCE PTC Therapeutics, Inc.
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