If I were sitting in front of the FDA I would ask if they had children. I would ask what their hopes and dreams were for their kids. Then I would ask, how they would feel knowing their child would not celebrate their 30th birthday. I've been on this journey with Andrew for 10 years. Ten years of creating a "new normal" after every loss. Ten years of watching a beautiful smart healthy young man grow so weak he can't do anything for himself. I would tell them of the heartache and anguish that we live with every day, in every way. How the stress and strain has taken the love and laughter out of our lives. I would tell them, Andrew does not have time! We don't have decades, years, or months for that matter.
When Andrew turned 16 in October, my prayer was to stabilize what he has left. His deletion set would be benefited by Eteplirsen. Would he be able to feed himself again, probably not, but would he be able to maintain what he currently has, it is hopeful. In ten years, Andrew has gone from a wonderfully active little boy, to a teenager trapped in his body. He has little hope and that has in itself has caused a number of issues. He feels like death is just waiting for him. This is a bright, funny, capable young man who has so much to offer this world, but is forced to watch as his friends get drivers permits, go dating, have parties, and think about a future. Andrew wants all the experiences in life a typical 16 yr old boy wants, I have begged and will continue to beg the FDA to reconsider, Andrew is on a death sentence, which is not of his doing! Before he loses one more dignity, I would invite a representative to come to our home, visit us, and I will show them why the risk would be worth it! We need hope, we need something that will change the course of this disease.
We need ACTION!