Duchenne Muscular Dystrophy Foundations Work Together to Fund Novel Therapy for Leading Genetic Killer of Young Boys.

DENVER -- Four Duchenne foundations, Charley's Fund, CureDuchenne, The Nash Avery Foundation and a private family foundation, are working together to aggressively fund research that could help extend the lives of thousands of boys worldwide afflicted with Duchenne Muscular Dystrophy (DMD). They have pooled their resources to fund a novel approach for a potential treatment being conducted by Dr. Brian Tseng, MD/PhD, a prominent muscular dystrophy researcher at the University of Colorado Health Sciences Center and a doctor at The Children's Hospital of Denver. Dr. Tseng's lab is testing the effect of a membrane sealant on the leaky dystrophin-deficient membranes of skeletal muscle cells. The purpose of this project is to develop an effective treatment that will strengthen the cell membranes, using a chemical called poloxamer 407, as a chemical Band-Aid, patching these holes and tears in the muscle.

"We are currently testing a compound called poloxamer 407 as a 'molecular band-aid' to patch muscle membrane leaks and evaluate if it can improve skeletal muscle integrity and function," said Dr. Tseng. "This direction was triggered by two high school students working in my lab, Lalith Polepeddi and Mike Polmear (Denver's own Whiz Kids). The original idea of using a smaller membrane sealant compound called poloxamer 188 for cardiac muscle was developed in Dr. Joseph Metzger's lab at the University of Michigan. Poloxamer 407 is thought to be a relatively safe compound. In fact, poloxamer 407 is found in daily household commodities such as mouthwashes, children's cavity rinses, suppositories and also used as the solid coating of many pharmaceutical pills and tablets. This study is significant because it could one day represent a therapeutic treatment designed to compliment and enhance other therapies for DMD. In essence, we are trying to help seal up the muscles' leaky membranes. This is not a cure and the research is very preliminary, but it is a direction that has not been explored before."

"We're not parent support organizations. Our foundations are focused on the business of finding and funding promising research and technologies that could help save our sons," said Debra Miller, President, CureDuchenne. "We're pooling our resources to maximize our effectiveness. We work with prominent scientists around the world, do substantial due diligence on new projects and work together to quickly fund those that have high potential to impact the lives of our Duchenne boys. We are passionate about our success, as each of us has a personal interest in making sure we make our deadline C* our sons."

About Duchenne Muscular Dystrophy (DMD)

Every year, an estimated 20,000 babies (almost all of them boys) are born with Duchenne Muscular Dystrophy, worldwide. Their muscle cells will die and not regenerate. They will be in wheel chairs by 12 and, historically, most have not survived their teenage years. An estimated 1 in every 3,500 boys is affected by DMD. It knows no boundaries and crosses all cultures and races. There currently is no cure, but for the first time there are promising therapies on the horizon.

About the Duchenne Foundations

Charley's Fund is a non-profit organization founded by Tracy and Benjamin Seckler. 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. For more information contact: Charley's Fund, PO Box 297, South Egremont, MA 01258. Visit our website at www.charleysfund.org. Call [phone] or [phone] (877-4-END-DMD) or email [e-mail].

CureDuchenne is a non-profit organization founded in 2003 by Debra and Paul Miller. Our vision is our name C* to cure Duchenne Muscular Dystrophy. CureDuchenne aggressively seeks out and funds research and expedites practical therapies that will help save THIS generation of Duchenne boys. For more information, contact: CureDuchenne, 3334 E. Coast Hwy. #157, Corona del Mar, CA 92625. Visit our website at www.cureduchenne.org. Call [phone] or email [e-mail].

The Nash Avery Foundation is a non-profit organization founded by Tom and Angie Wicka. Our Mission is to defeat Duchenne Muscular Dystrophy, so that one day soon these boys will live happy lives well beyond their twentieth birthdays. We will relentlessly push, prod and pound on any door in order to make people aware of the deadliness of DMD and to raise funds to cure these children. For more information, contact: The Nash Avery Foundation, 3109 West 50th Street, Suite 205, Minneapolis, MN 55410-2102. Call [phone] or email [e-mail].

For more information on Dr. Brian Tseng and the Denver "Whiz Kids," contact: TsengLab at www.uchsc.edu/sm/neuro/dmd/intro.htm or contact Brian Tseng, MD/PhD, at [e-mail]. View ABC's interview, High School Whiz Kids Search for MD Cure, at abcnews.go.com/GMA/Health/story?id=1808757.