We are Sarepta Therapeutics: a biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases. Our primary focus is on rapidly advancing Duchenne muscular dystrophy (DMD) therapies that are designed to treat the underlying cause of the disease.

OPTIMIZED TO GET TO THE HEART OF DMD

To get to the heart of DMD, it is critical for gene therapy to be delivered to skeletal and cardiac muscle.

Our goal is to develop therapies to treat 100% of individuals with this devastating disease.

Our commitment to the patients and families impacted by Duchenne muscular dystrophy extends beyond treatment. We are proud to offer SareptAssist™, a patient support program for those seeking information on therapy.

Our unique RNA-based technology has the potential to advance treatments in numerous areas of high unmet need. We are continuing to advance our clinical programs for Duchenne muscular dystrophy.

We seek collaborations with leaders in industry, government, and academia to make meaningful advances in medicine. Our proprietary and highly versatile RNA-based technologies form the foundation of our partnering strategy.