CureDuchenne Ventures

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. 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. In addition to research aimed at replacing the missing protein, CureDuchenne has backed research for anti-inflammatory and anti-fibrotic treatments, gene therapy as well as drugs to treat heart failure, and physical therapy and education to help keep patients ambulatory as long as possible. There may be no single cure for Duchenne. Rather, patients in the future may take a cocktail of drugs, much as people with AIDS do today. Also, visit us at: Facebook: Facebook.com/cureduchenne/Twitter: Twitter.com/CureDuchenneInstagram: Instagram.com/CureDuchenneMD