Sarepta Therapeutics is a biotechnology company that develops RNA-based therapeutics for the treatment of rare neuromuscular diseases. The company's pipeline includes a number of drugs that are in various stages of development, including: * SRP-9001, a small interfering RNA (siRNA) that is being developed for the treatment of Duchenne muscular dystrophy (DMD). SRP-9001 is currently in Phase 3 clinical trials. * SRP-5051, a small molecule that is being developed for the treatment of myotonic dystrophy type 1 (DM1). SRP-5051 is currently in Phase 2 clinical trials. * SRP-9002, a small molecule that is being developed for the treatment of facioscapulohumeral muscular dystrophy (FSHD). SRP-9002 is currently in Phase 1 clinical trials. * SRP-9003, a small molecule that is being developed for the treatment of limb-girdle muscular dystrophy type 2A (LGMD2A). SRP-9003 is currently in Phase 1 clinical trials. * SRP-9004, a small molecule that is being developed for the treatment of amyotrophic lateral sclerosis (ALS). SRP-9004 is currently in Phase 1 clinical trials. Sarepta Therapeutics is also developing a number of other RNA-based therapeutics for the treatment of other rare neuromuscular diseases. The company's goal is to develop treatments that will improve the lives of patients with these devastating diseases.