My story begins in 1980 when I became a camp counselor for MDA summer camp. From 1980 to 2012, I watched the campers enjoying being the "norm" for a week. I fell in love with their courage and their joy for life. I watched over the years as the disease ravaged their bodies, but not their spirits. It broke my heart each time we lost a camper.
Fast forward to March 18, 2013 in Guangzhou, China. This was "Gotcha Day" This is the day I met my son, Bobby. He had been diagnosed in China with Progressive Muscular Dystrophy, which I knew to be Duchenne from reading his file. I knew going in to the adoption, what he faced and had vowed when I first saw his picture that I would be there to go through it with him. No child should have to face Duchenne alone. In China, Bobby did not receive medication nor did they do stretches. I knew that bringing him home to the US would mean he would have access to top of the line medical care and that I would fight to get it for him. I believe in doing whatever we can to help find treatments to slow the progression and to ultimately find a cure. Bobby is taking part in a Natural History Study as well as the DMDimaging study. My hope is that the imaging study will be able to provide an alternate means of measurement to the 6 minute walk, as the walk severely limits the pool of boys that can participate in the drug trials. But the imaging trial will take time to complete. Time Bobby and the other boys with Duchenne don't have.
Every day brings challenges. Bobby has already lost strength since coming home in March. He bear crawls the stairs and when he is tired he has difficulty making it to the top. He uses a wheelchair for long distance and at school during transitions to conserve his energy. I will have to buy a new vehicle sooner than expected and will have to move and build an accessible home very soon.
I brought Bobby home with the intention of giving him every opportunity to live a longer and stronger life. Hearing that now the FDA wants more data and is not encouraging accelerated approval is very disheartening. This disease is like no other and those who have Duchenne cannot afford to wait. This is a fatal disease and as such many of us, as parents, are willing to weigh the risk and possible side effects of new drugs in trial. Realistically the viewpoint is try something that has the potential to slow down the progression and prolong life, or sit back and allow your child to die far too young.
I did not adopt my son and bring him to the US to sit back and watch him die. I am fighting for his life. Please help me in my fight and allow accelerated approval for the drugs that could save his life. I am doing my part and participating in studies that will allow more data to be collected. Please do your part. My son's life and the lives of all the boys with Duchenne depend on it.