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PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?

Executive Summary

US FDA appears willing to consider accelerated approval for PTC's Duchenne Muscular Dystrophy therapy ataluren if company is able to provide compelling evidence of increased levels of dystrophin in boys with nonsense-mutation DMD. That is quite a reversal from the agency’s view heading into PTC’s appeal. 
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