I am writing to share some wonderful news. My son Devin is walking, running, and can even ride a bike all by himself. That may not seem all that amazing on its own, but Devin’s circumstances are quite unique. Devin lives with Duchenne Muscular Dystrophy (DMD), a terminal muscle disease. It’s a disease that gradually takes away muscle function throughout the entire body until movement is no longer possible. Generally, most boys who have DMD do not survive beyond their mid-twenties, and those that do will be using a wheelchair by age 12.
Patient support organizations have always talked a lot about hope and for families like mine, that’s all that we’ve ever had… until recently. Devin was involved in a clinical trial over a period of 815 days. During that time, he made 111 round trips to Chicago and 8 round trips to Iowa totaling over 43,970 miles on the road. He endured 110 IV infusions, 3 surgeries, and countless hours undergoing a variety tests. Today at nearly 16 years old, Devin is still as strong as ever!
The FDA approved Exondys51 for commercial use on September 19, 2016. It became the first drug ever approved for treatment of Duchenne Muscular Dystrophy. The drug that created dramatic change in Devin’s life is now available to thousands of other boys who have been waiting in anticipation. The hope we’ve heard so much about was founded in confidence and I’m thrilled to say, has now been delivered!
Research that lead to development of Exondys51 was partially funded by grants from organizations in the duchenne community. These research grants would not be available without the generous donations provided by many very thoughtful people. Each donation holds value well beyond the individual dollar amount given. Each one holds limitless potential that may change the course of muscle disease forever, just as we’ve experienced with Exondys51. Families like mine recognize that possibility and I can say firsthand, that your support provides us a great deal of assurance that we are not in this alone. I’m eternally grateful for everyone that has made a contribution to help us fulfill this mission. Thank you all very much for your dedication to the organizations that represent many DMD families, just like mine. We truly appreciate everything you do to ensure a future free from muscle disease. You’ve given us a real reason to believe that anything is possible.