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 What We're Funding

CureDuchenne continues to leverage our effectiveness by partnering with other foundations to quickly fund viable research projects. CureDuchenne and three other Duchenne foundations have recently 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. This direction was triggered by two high school students working in Dr. Tseng’s 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. This is not a cure and the research is very preliminary, but it is a direction that has not been explored before.” For more information on Dr. Brian Tseng and the Denver “Whiz Kids”, visit: TsengLab at www.uchsc.edu/sm/neuro/dmd/intro.htm.  View ABC’s interview, "High School Whiz Kids Search for MD Cure," at  www.abcnews.go.com.   Watch ABC's video report, "Whiz Kids Big Muscular Dystrophy Find", at  www.abcnews.go.com/Video.

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