Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of diseases. The company is developing therapeutic candidates for a range of diseases and disorders, including Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophies, Mucopolysaccharidosis type IIIA and other neuromuscular and central nervous system. The company's commercial product, EXONDYS 51? (eteplirsen) Injection 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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