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FDA Refuses To File Translarna NDA; PTC Plummets

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Executive Summary

Just as things looked like they couldn't get any worse for the Duchenne muscular dystrophy (DMD) research and development community – and for patients – PTC Therapeutics Inc. revealed it had received a refuse-to-file (RTF) letter from the FDA for the firm's new drug application (NDA) for Translarna (ataluren).

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