"Lucky" isn't a word that usually comes to mind when thinking about children with Duchenne. The disease devastates kids and their familie...
"Lucky" isn't a word that usually comes to mind when thinking about children with Duchenne. The disease devastates kids and their families. It is 100% fatal. But, in a way, for the first time, we are fortunate. We have known what causes Duchenne for over 20 years. Affected children do not produce a crucial protein called Dystrophin. As a result, their muscles break down and they lose mobility and function and, ultimately, their lives. Great minds in science have devoted their lives to cracking the code on Duchenne. For decades, there have been necessary failures, massive community advocacy, grassroots and government funding, and numerous clinical trials. It has been a long and grueling marathon. But now, with a real potential treatment waiting for FDA approval, it has become a sprint. A race to get this and future therapies to patients. A Race to Yes.