My story began many years ago when my baby brother was diagnosed with Duchenne MD at the age of 4. I was six and we had two older sisters. My parents were shocked. They had never heard of this rare disease. The doctors didn't know what to do and offered no where to turn. They drove many hours each time to a children's hospital two states away to see a neurologist, a physical therapist and to have the surgeries done that would hopefully help. That was over 30 years ago and boy have we come a long way. My brother died when he had just reached his 20th birthday and I cannot even begin to describe the impact of losing him and the grief that my family felt...fast forward a little, my husband and I married about two years later and started our family. I am a carrier of DMD and we had two healthy girls and a baby boy. At the age of two weeks old, my newborn son was tested and there we stood facing the same reality that my parents had faced 3o years prior.
Our son is now four years old, and he has hope right here at his fingertips. My husband and I are so thankful for this hope because we know exactly what is ahead for him if there is no intervention. We know that we will bury our son. We know the effects it will have on us and our marriage, on his sisters and their lives, on his friends, his grandparents, cousins, teachers, his community. We know. We have lived this before. But I mentioned that we do have hope...a new hope that my parents and my brother did not have. There is actually something that exists that will help our son, REALLY help him. Maybe we can have dreams for him again that he may play a team sport or be able to run and jump and climb trees and ride a bike and play army man in the woods with his buddies and go to school on his own two feet instead of in a wheelchair by the 5th grade. We have hope for these things and we are excited about it. But we are scared, too. We know it may get to be too late for him.
We pray that we can get this drug approved, or get in a trial, or have it be available somewhere in this world and we will go there and get it! We will do what it takes to save our son and to help others in his same situation, and help this generation of DMD boys so maybe this can be a disease that one day no one will ever have to know or worry about. We pray that this will be the first generation of DMD boys to live, instead of the last generation to die.