Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company, which is engaged in the discovery and development of therapeutics for the treatment of rare diseases. The company is headquartered in Cambridge, Massachusetts and currently employs 1,372 full-time employees. The firm is focused on helping patients through the discovery and development of ribonucleic acid (RNA)-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. The company has developed multiple approved products for the treatment of Duchenne muscular dystrophy (Duchenne) and is developing potential therapeutic candidates for a range of diseases and disorders, including Duchenne, Limb-girdle muscular dystrophies (LGMDs) and other neuromuscular and central nervous system (CNS) related disorders. The firm has developed and commercialized four approved products for the treatment of Duchenne: EXONDYS 51 (eteplirsen), Injection (EXONDYS 51), VYONDYS 53 (golodirsen) Injection (VYONDYS 53), AMONDYS 45 (casimersen) Injection (AMONDYS 45), and ELEVIDYS. Its pipeline includes over 40 programs at various stages of discovery, pre-clinical and clinical development.
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