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Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases.
Research focus: Exon Skipping.
Status: Accelerated approval of EXONDYS 51 (eteplirsen) by FDA. Other candidates for exon skipping in various stages of clinical development.
Website: https://www.sarepta.com
“CureDuchenne helped us very early on in in the development of the program. They gave us a jump start on clinical trial development. This is a very long journey. I would look at eteplirsen as the first step in a long process of trying to get better and better drugs for this community.” — Ed Kaye, M.D., CEO and Chief Medical Officer of Sarepta.
