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Company Description (as filed with the SEC)
We are a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. Applying our proprietary, highly-differentiated and innovative platform technologies, we are able to target a broad range of diseases and disorders through distinct RNA-targeted mechanisms of action. We are primarily focused on rapidly advancing the development of our potentially disease-modifying DMD drug candidates, including our lead DMD product candidate, eteplirsen, designed to skip exon 51. On August 25, 2015, we announced the FDA filing of our NDA for eteplirsen for the treatment of DMD amenable to exon 51 skipping. The FDA postponed the Advisory Committee meeting for the review of the eteplirsen NDA previously scheduled for January 22, 2016 due to severe weather. ... More ...
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|Annual EPS Est:||$-5.2|
|Quarterly EPS Est:||-1.34|