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Last Trade
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Last Trade:
$116.64
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Change:
-2.09 (-1.76%)
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Trade Time:
04:00 PM EST
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Market Cap:
$8.59B
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Description of Business
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We are a commercial-stage biopharmaceutical company focused on helping patients
through the discovery and development of unique RNA-targeted therapeutics, gene
therapy and other genetic therapeutic modalities for the treatment of rare
diseases. Applying our proprietary, highly-differentiated and innovative
technologies, and through collaborations with our strategic partners, we are
developing potential therapeutic candidates for a broad range of diseases and
disorders, including Duchenne muscular dystrophy ("DMD"), Limb-girdle muscular
dystrophies ("LGMDs"), Mucopolysaccharidosis type IIIA ("MPS IIIA") and Pompe.
Our first commercial product in the U.S., EXONDYS 51® (eteplirsen) Injection
("EXONDYS 51"), was granted accelerated approval by the FDA on September 19,
2016. EXONDYS 51 is indicated for the treatment of DMD in patients who have a
confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
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