On the recordJuly 25, 2013
It's an honor to talk about some courageous children who are changing the way that we think about Duchenne Muscular Dystrophy. It affects nearly 20,000 babies a year in the United States, robbing them of the muscle development they need to grow into a healthy childhood. These children, like Gabe Griffin of Birmingham, who you see in this photo, are full of strength, spirit, and hope. They inspire all of us. As he grows into adulthood, his muscle development will be arrested if we don't make progress. Thanks to research and advocacy by parents like Gabe's, Scott and Traci Griffin, as well as Joel and Dana Wood, here in Washington new treatments are being developed for Duchenne. But for families, the progress needs to come faster. The FDA is now considering whether to grant accelerated approval to a potential breakthrough therapy. It's a drug called Eteplirsen. While properly taking safety into account, it is important for the FDA to make a timely decision on this drug. When you look at this picture, you know that we must do everything possible to help these amazing young people to enjoy the happy and healthy childhood that so many of us were blessed with. Let's do everything we can to urge the FDA to research this drug and make it available to the general public. ____________________
