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PTC Therapeutics Announces Additional Study of Ataluren in Patients with Advanced Nonsense Mutation Duchenne/Becker Muscular Dystrophy
Date:1/19/2010

rnalLink');" target='_blank' href="http://www.mda.org/">www.mda.org) or Parent Project Muscular Dystrophy (www.parentprojectmd.org).

ABOUT ATALUREN (PTC124®)

Ataluren is the first investigational new drug designed to restore the production of a functioning protein in patients with genetic disorders due to a nonsense mutation.  A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein, such as dystrophin in the case of nmDBMD.  Ataluren is currently being investigated for use in patients with nonsense mutation Duchenne and Becker muscular dystrophy (nmDBMD), nonsense mutation cystic fibrosis (nmCF) and nonsense mutation hemophilia A and B (nmHA/nmHB).  Ataluren has been granted orphan drug status for the treatment of nmDBMD and nmCF by the U.S. Food and Drug Administration (FDA) and the European Commission.  The FDA has also granted ataluren Subpart E and Fast Track designation for expedited development, evaluation, and marketing.  The development of ataluren has been supported by the FDA Office of Orphan Products Development, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), and the National Center for Research Resources.

COLLABORATION WITH GENZYME

PTC Therapeutics has an exclusive collaboration with Genzyme Corporation for the development and commercialization of ataluren.  PTC Therapeutics will commercialize ataluren in the United States and Canada, while G
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SOURCE PTC Therapeutics, Inc.
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