Disease Areas

At Sarepta, we’re committed to pursuing some of the world’s most debilitating, prevalent, and complex rare genetic diseases. Today, our primary focus is on Duchenne muscular dystrophy, limb-girdle muscular dystrophies, Mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome A, and Charcot-Marie-Tooth disease.


Sarepta's Patient Affairs Team

Patient Affairs team

Each member of our Patient Affairs Team brings passion, energy, and a deep commitment to their work every day whether they hail from our U.S. headquarters in Cambridge, Massachusetts, or one of our locations in Europe. 

Corporate Grants and Giving

Serving the rare disease community goes beyond developing precision genetic medicines. Corporate Giving is a core part of Sarepta’s mission.