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Rare Candace

An unconventional disruption of rare disease.

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Tag Archives: Duchenne

A Winning Day for Orphan Drugs!

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This morning, the Food and Drug Administration announced that it granted accelerated approval to Sarepta Therapeutics’ drug Eteplirsen. This comes after months of waiting for a decision after an extremely intense Adcomm meeting.  Continue reading →

Posted on September 19, 2016 by RareCandace. | Tagged DMD, Duchenne, Eteplirsen, orphan drugs, Sarepta

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