PTC Therapeutics Announces $25 Million Award From Cystic Fibrosis Foundation Therapeutics for Development...( _new http://www.cff.org ). About PT...),Health

About PTC124

PTC124 is an orally delivered investigational new drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, preventing production of a functional protein. In Phase 2a clinical trials in nonsense-mutation-mediated cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD), PTC124 has demonstrated the ability to produce protein across a variety 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 in DMD with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mutation-mediated genetic disorders may provide clinical benefits.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation, the leading organization focused on curing and controlling cystic fibrosis, has invested nearly $300 million in drug research with biotech companies since 1998 to develop therapies to fight CF. As a result, the Foundation has built a drug pipeline with more than 30 promising therapies in development. Virtually all the approved CF therapies available today were made possible because of the support of the Foundation. Based in Bethesda, MD, the Foundation has 80 chapters and branch offices, and supports and accredits a nationwide network of 115 CF care centers that provide treatment and vital resources to patients and families. For more information visit http://www.cff.org

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-transcriptiona
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