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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

s ground-breaking study in DBMD patients who have lost the ability to walk," said Valerie Cwik, M.D., Executive Vice President -- Research and Medical Director, Muscular Dystrophy Association.  "This trial underscores the importance of MDA's translational research effort.  In April 2005, MDA awarded an initial $1.5 million grant to PTC in support of early clinical development of ataluren in nmDBMD.  Now we have a trial that will provide valuable information on older DBMD boys and young men that will assist in the design of future clinical trials."

This one-year, Phase 2a study is evaluating the safety, pharmacodynamic activity, and pharmacokinetics of ataluren, an investigational new drug, while assessing the use of several outcome measures of physical, pulmonary, and cardiac function in patients with advanced disease.  Approximately 30 patients are being enrolled in the trial at five sites in the United States, which are part of the MDA's DMD Research Network, and at one site in the United Kingdom.  Additional information on this study can be found on http://clinicaltrials.gov.

"We are eager to participate in this innovative study of non-ambulatory DMD patients," stated John W. Day, M.D., Ph.D., principal investigator at the University of Minnesota's Paul and Sheila Wellstone Muscular Dystrophy Center.  "This precedent-setting trial is an important extension of current studies of younger, ambulatory boys with DMD, and will help define methods
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SOURCE PTC Therapeutics, Inc.
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