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Agony & Ecstasy Of Sarepta: A New FDA Rare Disease Standard?

Executive Summary

Even though investors viewed the FDA's willingness to give Sarepta another chance to provide more data as a positive step, most analysts said it was simply delaying the inevitable of rejecting the firm's Duchenne muscular dystrophy drug eteplirsen. Others have cautioned the FDA may be setting a new standard for approving rare disease drugs, in which other companies may demand similar attention.


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