PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular...(ng and life-threatening disorder. St...),Health

Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics added, "Initiation of the Phase 2b trial is an important milestone for PTC. The trial builds on the results we have achieved to date in DMD and cystic fibrosis (CF) and constitutes a major step forward in establishing the potential for PTC124 as a paradigm shift in the treatment of genetic disorders. Our future plans for PTC124 include the initiation of longer-term studies in CF, as well as additional proof-of-concept studies in other indications."

About Duchenne and Becker Muscular Dystrophy

Duchenne and Becker muscular dystrophy (DMD/BMD) are progressive muscle disorders that cause the loss of both muscle function and independence. DMD/BMD 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). It is estimated that one in 10 DMD patients are likely to have a Becker presentation, a milder form of the disease that is associated with later manifestation of symptoms. In essence, DMD and BMD represent a continuum of the same disease. More information regarding DMD and BMD is available through the Muscular Dystrophy Association (http://www.mdausa.org), the Parent Project Muscular Dystrophy (http://www.parentprojectmd.org), and the Association Francaise contre les Myopathies (http://www.afm-france.org).

About PTC124

PTC124 is an orally delivered investigational new drug in Phase 2 clinical development for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely halt the translation process
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