Kyle and I are headed to Washington DC in just a few weeks to attend the FDA Advisory Committee meeting to consider the first DMD drug- eteplirsen.
We love our little superhero so much and will do everything in our power to help him live a longer and better life. His two year old dream right now is to "run fast like batman". Without treatment, he will never be able to run in his lifetime.
This drug will not actually benefit John Michael but if approved will pave the way for more drugs that will help his particular deletions. FDA, PLEASE APPROVE!