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PTC Therapeutics/Genzyme's ataluren fails in Phase IIb muscular dystrophy trial

This article was originally published in Scrip

Executive Summary

PTC Therapeutics and Genzyme's ataluren, an experimental nonsense mutation suppressor, has failed in nonsense mutation Duchenne/Becker Muscular Dystrophy (nmDBMD), show top-line Phase IIb results. Around 10-15% of DBMD cases are due to nonsense mutations.



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