Sarepta is at the forefront of precision genetic medicine, with over 40 therapies in various stages of development. Our disease areas include Duchenne muscular dystrophy (DMD), six Limb-girdle muscular dystrophies (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA (Sanfilippo Syndrome type A), and other CNS-related disorders. The company’s programs and research focus span several therapeutic modalities, including gene therapy, RNA technologies, and gene editing.