Form Type:  D
Filing Date:  2/28/2020 
CIK:  0000873303 
Address:  215 FIRST STREET
City, State, Zip:  CAMBRIDGE, Massachusetts 02142 
Telephone:  617-274-4000 
Fiscal Year:  12/31 
Last Trade
Last Trade: 
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Trade Time: 
Jun 11  
Market Cap: 
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Description of Business
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") 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. 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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