Understanding Duchenne

Duchenne muscular dystrophy is a progressive muscle disorder that affects primarily boys, causing the loss of both muscle function and independence. It is the most common of the 20 muscular dystrophies and is the number one fatal genetic disorder diagnosed during childhood today. Nearly all boys with Duchenne die by the age of 20 from respiratory and/or cardiac complications.
The facts about Duchenne:

• Approximately 20,000 children worldwide, or one in every 3,500 boys, are born with Duchenne. A boy inherits Duchenne when he receives an X chromosome that fails to make the protein dystrophin, an essential building block of healthy muscle.
• Boys with Duchenne show signs of muscle weakness as early as age 3.
• Walking, running, or riding a bike is a challenge for a boy with Duchenne, as the disease gradually weakens the skeletal or voluntary muscles in the arms, legs and trunk. Nearly all boys with Duchenne lose the ability to walk sometime between ages 7 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.
• There is no way to stop or reverse the muscle degeneration of Duchenne. Accepted treatments can only lessen symptoms and improve the quality of life.
• The only significant breakthrough in Duchenne research was the discovery of the defective gene causing Duchenne – dystrophin – in 1986 at the Children’s Hospital in Boston, MA.