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Last Trade
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Last Trade:
$151.00
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Change:
1.24 (0.83%)
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Trade Time:
Jun 02
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Market Cap:
$11.78B
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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 other
neuromuscular and central nervous system ("CNS") related disorders.
Our first commercial product, EXONDYS 51 (eteplirsen) Injection ("EXONDYS 51"),
was granted accelerated approval by the U.S. Food and Drug Administration
("FDA") on September 19, 2016.
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