Duchenne muscular dystrophy is the disease you could help cure. The question is, can we do it in time to save Charley and others in this generation? Duchenne is the most common fatal genetic disease to strike children around the world. Kids with DMD cannot produce dystrophin, a protein necessary for muscle strength and function. As a result, every skeletal muscle in the body deteriorates. To date, there is no approved treatment or cure.
BUT…scientists discovered what causes Duchnne back in 1987, and finally the stars have aligned for the disease to be defeated. Several drug companies and investors are putting resources toward developing new treatments. Entrepreneurs and scientists are coming up with better ways to attack the pathology. Nonprofit foundations are helping to smooth the road and finance the best research. Momentum has never been more powerful. Now is our time.
The Facts About Duchenne
What is it?
- 100% fatal, with most kids losing their lives in their late teens or early twenties
- Most boys with DMD are in a wheelchair between 10-12 years old
- Associated with respiratory failure, heart failure, and debilitating orthopedic complications
- There are 9 types of muscular dystrophy, and Duchenne is the most severe
Who Gets It?
- Approximately one in 3,500 boys born the world over
- In very rare cases, a girl is affected (approximately one in a million)
- An estimated 15,000-20,000 children in the US
- Approximately 200,000 around the world
- DMD affects boys of every ethnicity and geographic location
- One third of all DMD cases are the result of random in utero mutation, with no warning before the affected baby is born
What Can Be Done?
- Steroids have been shown to delay the progression of the disease.
- Steroids are often associated with serious side effects.
- Much is still unknown about how steroids work.
- Despite daily stretching, many DMD boys need orthopedic braces and corrective surgery to combat debilitating muscle contractures.
- Because there is no acceptable universal treatment for Duchenne, many families turn to alternative therapies (nutritional supplements, vitamins, acupuncture).