Sarepta Therapeutics, Inc.

Sarepta Therapeutics develops medicines for rare genetic diseases, particularly Duchenne muscular dystrophy, a serious condition that affects muscle function and is caused by problems in a specific gene. The company's approved drugs, including EXONDYS 51, VYONDYS 53, and AMONDYS 45, work by using RNA technology to help patients' bodies produce a partial version of the missing protein their muscles need. Sarepta also works on gene therapy treatments, which aim to fix the genetic problem itself rather than just treat the symptoms, and has research programs for other rare genetic muscle diseases.