I want the FDA to know that when Duchenne enters your life, there is no "son, you can grow up and be anything you want to be" discussion anymore. I want them to know that it changes everything about parenting you ever thought you knew to be true. Before Duchenne, I pushed my children to do their best at school, and set goals for themselves. Give 100%, isn't that what we're supposed to do? But just how important is education for a child that may not live long enough to utilize it?? How important is discipline when by the time my son reaches the age where he can start to actually reflect on and practice our teachings to him, he will be struggling just to breathe and eat??
I made a commitment to him when he was diagnosed - I will knock down every door. I will raise as much money as I can. I will search the world over for any opportunity to save him. I will give up my career in order to focus on this goal full-time. I will give up every single personal goal I have for myself so that he can have what he needs/wants from me. After all, God chose me to be his mother for a reason, and I understand that he is a gift for which I will always be grateful.
I want the FDA to consider the possibility of not only acting as a regulator, but as a facilitator. A partner. While we recognize the need for safety and appreciate their efforts, our fight is to save our sons' lives. NOW. I would love to see the FDA put a panel together that interacts with our community about clinical trial design. It was apparent from last year's PPMD Conference there is a disconnect between regulator and clinician regarding endpoints. The FDA says "we review what we are given" and the clinician says "we want to know what is an acceptable endpoint and when we ask we get no information"...why can't there be more of a partnership here? The FDA can be our true heroes here.
No other country has approved a treatment for this God awful disease yet. Our country can show true leadership and compassion by accelerating the process for approval and partnering with us to get this done.