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Targeting Specific Mutations Reveals Promising New Approach to,Treating Genetic Disorders; Preclinical Data Published in Nature

dons and the restoration of the production of full-length, functional proteins. In Phase 1 clinical trials, PTC124 was generally well tolerated, achieved target plasma concentrations that have been associated with activity in preclinical models, and did not induce readthrough of normal stop codons. PTC is currently conducting Phase 2 clinical trials of PTC124 in nonsense-mutation-mediated cystic fibrosis and Duchenne muscular dystrophy.

The FDA has granted PTC124 Fast-Track designations and Orphan Drug designations for the treatment of CF and DMD due to nonsense mutations. PTC124's development is supported by grants from the Muscular Dystrophy Association (MDA), Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), Parent Project Muscular Dystrophy (PPMD), FDA's Office of Orphan Products Development (OOPD), the National Institutes of Health, and by General Clinical Research Center grants from the National Center for Research Resources (NCRR).

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally-discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-molecules (GEMS) technology platform, which has been the basis for collaborations with leading pharmaceutical and biotechnology companies such as Pfizer, CV Therapeutics, and Schering-Plough.

CONTACT: Jane Baj of PTC Therapeutics, Inc., +1-908-222-7000, Ext. 167,, or Sheryl
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