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PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy
Date:2/3/2009

r the next year as we continue to work to develop additional innovative therapies for the muscular dystrophy community."

About DMD/BMD

Duchenne and Becker muscular dystrophy (DMD/BMD) are progressive muscle disorders that result in the loss of both muscle function and independence. DMD/BMD is perhaps the most prevalent form 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. Further information regarding DMD and BMD is available through the Muscular Dystrophy Association (www.mdausa.org), Parent Project Muscular Dystrophy (www.parentprojectmd.org) and the Association Francaise contre les Myopathies (www.afm-france.org).

About Ataluren (PTC124(TM))

Ataluren is an orally delivered, investigational new drug discovered by PTC Therapeutics. The drug is being developed as a new approach for the treatment of nonsense mutation genetic disorders. Nonsense mutations create a premature stop signal in the mRNA causing the ribosome to terminate translation before a full-length protein is generated. This causes the protein to be truncated and non-functioning. Ataluren is designed to allow the ribosome to continue translation of the mRNA, by overriding the premature stop signal leading to the formation of a functioning protein. Ataluren is a potential therapy for patients with nonsense mutation genetic disorders.

Ataluren has demonstrated proof of concept in Phase 2a clinical trials and is currently
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SOURCE PTC Therapeutics, Inc.
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