Sarepta Therapeutics is a biopharmaceutical company focused on developing and commercializing therapies for rare genetic diseases, particularly Duchenne muscular dystrophy (DMD). They are known for developing exon-skipping therapies, which aim to address specific genetic mutations in DMD patients. Sarepta's research and development efforts also extend to other neuromuscular and central nervous system disorders, employing gene therapy and RNA-based approaches to target the root causes of these diseases.
