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Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare diseases. Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in Limb-girdle muscular dystrophy (LGMD), Charcot-Marie-Tooth (CMT) and CNS-related disorders, reaching a total of over 20 therapies in various stages of development. The Company`s programs span across several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is poised to be the most meaningful precision genetic medicine company in the world and make a profound difference in the lives of patients suffering from rare neuromuscular diseases and other rare diseases.
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