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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

SOUTH PLAINFIELD, N.J., Jan. 19 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently.  This trial is evaluating the best methods for measuring functional abilities in patients who have lost independent mobility.  Patients with nmDBMD develop progressive muscle weakness that leads to deterioration of ambulation, wheelchair dependency, progressive loss of upper limb strength, and eventual respiratory and cardiac failure.  The study, which is expected to complete enrollment rapidly, is being funded in part by a $1 million grant from the Muscular Dystrophy Association, and will involve MDA's five-center DMD Clinical Research Network and a site in the United Kingdom.  

(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )

"Given the relentlessly progressive course of DBMD and its impact on multiple organ systems, we believe patients could benefit from therapeutic interventions at all stages of the disease.  This trial is an important step in understanding the best methods for evaluating new clinical treatments for these boys and young men," stated Langdon Miller, M.D., Chief Medical Officer at PTC.  

"The Muscular Dystrophy Association is proud to provide $1 million in funding towards thi
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SOURCE PTC Therapeutics, Inc.
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