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FDA Refuses To File Translarna NDA; PTC Plummets

This article was originally published in Scrip

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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