Creating value through cutting-edge science and an unwavering commitment to patients
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At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA and other CNS-related disorders, totaling over 40 therapies in various stages of development. Our programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing.
Our work is purposeful, audacious, urgent. Our commitment to do well by patients is unyielding as we build our position as the world's leader in precision genetic medicine.
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