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CureDuchenne – LinkedIn

Over the past decade, CureDuchenne has developed a multidimensional approach to finding and developing potential cures and treatments for Duchenne muscular dystrophy. Duchenne can result from a number of genetic variants, and because of the complexity of the human genome, a variety of approaches are needed to develop the most effective treatments.

So far, CureDuchenne has leveraged more than $100 million from biotech and pharmaceutical companies and other foundations to fund research leading to a cure. And while the ultimate goal is to find cures, CureDuchenne has been working in parallel to “treat the whole disease,” with multiple therapeutic strategies working together to deal with the many effects that Duchenne has on the body. Without the protein dystrophin, the body may develop fat, scar, or fibrous tissue where it would normally build muscle. Some of the most serious damage can be to the heart muscle. Cardiac failure is the leading cause of death for those with Duchenne.