At Sarepta, we are working with urgency to develop breakthrough therapies to treat genetic diseases. Currently, we have more than 40 investigational therapies in various stages of development—many already in late-stage clinical trials. In many cases, development is being accelerated by our gene therapy engine, which potentially provides a more efficient model for drug design.
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy, sometimes shortened to DMD or just Duchenne, is a rare genetic disease. It predominantly affects males, but, in rare cases, can also affect females. Duchenne causes the muscles in the body to become weak and damaged over time and is eventually fatal. The genetic change that causes Duchenne—a mutation in the DMD gene—happens before birth and can be inherited, or new mutations in the gene can occur spontaneously. Muscle weakness becomes increasingly noticeable between the ages of 3 and 5, and most patients use a wheelchair by the time they are 12. During adolescence, heart and breathing muscles weaken, leading to serious, life-threatening complications.
Duchenne affects approximately 1 in 3,500 to 5,000 males born worldwide.
Genetic Testing is a critical step in diagnosing any genetic disease. Decode Duchenne provides free genetic testing, interpretation, and counseling to people with Duchenne or Becker muscular dystrophy who meet certain eligibility criteria and who have been unable to access genetic testing in the past due to financial barriers, such as cost or lack of insurance coverage. Sarepta is a proud sponsor of Decode Duchenne, a program of Parent Project Muscular Dystrophy that is administered by Duchenne Connect.