Our battle with Duchenne began in November 2010 when our son, Isaac, just 13 months old at the time was diagnosed with this disease. Our family to that point has never even heard of Duchenne, nor had anyone in our family ever been diagnosed with any type of Muscular Dystrophy. On that day we also learned that there were no treatments available to treat the actual disease, only to treat symptoms of the disease. Then the doctor gave us the most life changing information, Duchenne is terminal! As I sat there holding this little baby of ours and looked into his beautiful eyes I could not for a single second let the thought that he would be taken by this disease. On this day, our family vowed to fight this disease with everything we have and now three years later we are fighting every day, every hour, every minute and every second.
To most of those at the FDA you would see the medical side of this disease. You would see how it affects the body and all of the different phases. What you don't see is the actual person on a daily basis struggling nor do you see the family coping with the many, many aspects of Duchenne. Duchenne affects every corner of your life. It affects relationships, it affects finances, it affects our health, it is figured into nearly every decision we make as a family. Duchenne even affects how my son attends preschool. Climbing steps is very difficult for him and so we had to chose a school with an elevator so he can go between floors like other children. His teachers pay close attention to him on the playground because he is more susceptible to fall, lose his balance etc. Duchenne affects our older daughter too. Things she thinks her little brother should be able to do that her friends younger siblings can are not possible for Isaac. She is required to be more patient about everything as we tend to Isaac's needs but also as we tend to fundraising and helping organizations like Parent Project MD or CureDuchenne.
When Isaac was diagnosed our family was informed about several drugs that were in trial two of which were extremely important, eteplirsen and drisapersen. These two drugs would aim to treat the actual disease and not the symptoms of Duchenne. Isaac is not a match for this gene mutation in which these two drugs would treat however, we knew if they were to get approval something to treat our son would be in his future. Approval from the FDA isn't something that we feel should just be handed out to anyone and everyone. We completely understand this. However, we do feel those at the FDA need to be better educated in these diseases and how they not only affect the actual body but also how they affect the families (everyone in the family) who is dealing with them. They need to understand all aspects, including the health of that child.
This is our story, the Clark Family!
Sincerely, Lisa Clark