This information is taken directly from the official Charley’s Fund website. Please visit the site at http://www.charleysfund.org to learn more and to make donations directly.
We direct money into the hands of researchers who have the best shot at developing a treatment or cure for Duchenne Muscular Dystrophy. Our goal is to cure DMD in time to save Charley’s life and the lives of thousands of boys like him.
Historically, most of the funds raised for Duchenne have subsidized the tremendous cost of caring for children with this brutal disease, not research to cure it. Several established organizations provide helpful services for families struggling to provide for their DMD-afflicted children. In contrast, our sole mission is to fund a cure or treatment for Duchenne. We invest our money in translational research – research that focuses on moving science from the lab into human clinical trials.
Duchenne Muscular Dystrophy is the most common fatal genetic disorder to affect children around the world. Children with DMD cannot produce dystrophin, a protein necessary for muscle strength and function. As a result, every skeletal muscle in the body deteriorates. Although Duchenne is the most common fatal genetic disorder to affect children, there is no cure.
For the moment, Charley is an active, athletic child who adores baseball, soccer, and snowboarding in the backyard. While the disease has yet to show its devastation, the stark facts about DMD are hard to believe:
- DMD has a 100% fatality rate.
- Most DMD kids die in their late teens or early twenties.
- Most boys with DMD are in a wheelchair, most between 10-12 years old.
- DMD is associated with respiratory failure, heart failure, and debilitating orthopedic complications.
Families all over the world are suffering from the effects of Duchenne, the most severe form of muscular dystrophy.
- Approximately one in 3,500 boys is born with DMD.
- An estimated 15,000-20,000 children in America are afflicted.
- 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.
Although much is known about the cause and effects of DMD, the current treatment options are extremely limited. Doctors recommend steroids and daily stretching, but these approaches are inadequate.
- 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).
To learn more about the disease and what causes it, visitThe Muscular Dystrophy Association, a large charity that supports 19 different neuromuscular diseases.
Another thorough explanation of the disease and its causes can be found at Parent Project Muscular Dystrophy, an organization that supports advocacy, education and research for Duchenne.
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