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Last Trade
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Last Trade:
$121.13
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Change:
-4.33 (-3.45%)
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Trade Time:
03:18 PM EST
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Market Cap:
$8.09B
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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 medicine approaches for the treatment of rare
neuromuscular diseases.
Applying our proprietary, highly-differentiated and innovative RNA-targeted
platform technologies, we are able to develop candidate therapies for a broad
range of diseases and disorders.
Our first commercial product in the U.S., 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.
A summary description of our product and main product candidates is as follows:
* EXONDYS 51, our first product, uses our PMO chemistry and exon-skipping
technology to skip exon 51 of the dystrophin gene.
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