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PTC Therapeutics Announces Data from Additional Clinical Studies of PTC124 in Cystic Fibrosis Confirming Activity
Date:6/12/2008

ariety of nonsense mutation types. Across all clinical studies to date, PTC124 has been generally well tolerated and has achieved target plasma concentrations that have been associated with activity in preclinical models. PTC124 is currently in Phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mediated genetic disorders may provide clinical benefits.

About PTC Therapeutics Inc.

PTC is a biopharmaceutical company focused on the discovery, development and commercialization 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, Celgene, CV Therapeutics and Schering-Plough. For more information, visit the company's website, http://www.ptcbio.com.


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