Duchenne Muscular Dystrophy
- A progressive muscle disorder that affects only boys
- The number one genetic killer in the world
- Causes the loss of both muscle function and independence
- Most common of the over 30 diseases of the muscles
- Nearly all boys with DMD die by the age of 20
- A boy inherits DMD when he receives an X chromosome that fails to make the protein dystrophin, an essential building block of healthy muscle
- Approximately 20,000 children worldwide, or one in every 3,500 boys, are born with DMD
- Boys with DMD show signs of muscle weakness as early as age 3.
- Walking, running, or riding a bike is a challenge for a boy with DMD, as the disease gradually weakens the skeletal or voluntary muscles in the arms, legs and trunk
- Nearly all boys with DMD lose the ability to walk sometime between ages 8 and 12 and require full-time use of a wheelchair
- By the early teens or even earlier, the disease may also affect the boy’s heart and respiratory muscles
- Accepted treatments, such as a steroid regimen can only lessen symptoms and improve the quality of life
- Steroid regimens wreak havoc on these boys’ bodies
- The only significant breakthrough in DMD research was the discovery of the defective gene causing DMD – dystrophin – in 1986 at the Children’s Hospital in Boston, MA
- In the 20 plus years since that discovery there is nothing to stop or reverse the muscle degeneration of DMD.