Sarepta Therapeutics

Cambridge,  MA 
United States

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 dystroph (LGMD), Charcot-Marie-Tooth (CMT) MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development.