While I know the impending winter storm precipitated your postponement of the Adcomm meeting scheduled for January 22nd, I implore you to reschedule it as soon as possible. So many families had already traveled to DC or were on their way prior to the meeting being postponed. These families and the rest of the Duchenne community know that our children do not have the time for further delays. Duchenne does not stop it’s progression to wait for postponements, delays or for that matter any reason. It is relentless in its continued deterioration of our children.
Today was a day full of reminders that my son has limited time. Today was the 100th day of school. He was to dress as what he would look like when he was 100 years old. While I realize not many of us actually make it to our 100th birthday, those with Duchenne currently have zero chance of reaching that age. So in thinking again I realized that my sweet 9 year old son, statistically speaking, is considered middle aged when looking at the average life expectancy for those with Duchenne. So maybe his costume today wasn’t all that off. His 100, statistically speaking, would then be somewhere in his mid to late 20’s.
My family’s story is a bit different than most families dealing with Duchenne. I started out as a volunteer summer camp counselor for MDA in 1980 and continued through 2012. I met and lost so many campers/friends over those years. I saw what Duchenne does, but there was always hope as the research was ongoing. In 2012 I decided to adopt a child. I saw my son’s picture on my agency's website and saw his diagnosis and knew that he was my son. I could not let him face Duchenne alone. I knew he would most likely not receive any treatment in his birth country due to his orphan status and their lack of knowledge about neuromuscular diseases.
Eteplirsen will not help my son, but it will help others and it will help pave the way for exon skipping 53 that will help my son. I adopted my son in order to provide him a family to help support him through this disease and because I knew that medical care and research in the US was one of his best chances to have a better quality and longer life. Please don’t prove that wrong. Our community understands that it will most likely take a cocktail of drugs to ultimately provide a cure. Anything that can slow the progression and allow those with Duchenne to live longer and stronger will give them a better shot at being around for the cure.
Right now there isn’t a treatment available. For a child who produces no dystrophin to produce any amount of dystrophin is a big deal. There have not been safety issues or detrimental side effects. Please listen to those who have been going through the drug trials. Look at them and see the children that have been able to have access to Eteplirsen.
Just because I knew before adopting my son that he had Duchenne and all about the devestation of DMD does not mean that watching his progression doesn't break my heart daily. It also doesn't mean that I resigned myself to sit back and watch that happen. On the contrary, I got to skip the initial diagnosis shock stage and start instead in the fight for my son's life stage.
Our country is a great country and my son is now a proud American. Please allow my son a chance to grow old. Please don’t make his birth country regret adopting to an American family instead of a family from another country who may allow him access to drugs that will help slow or halt the progression of his Duchenne.
I hold out hope that you will consider all the positives seen in the drug trials(no matter how small you may think they are, as to those of us fighting the fight against the villianous Duchenne, small victories are huge!) I count on you to do the right thing and allow Eteplirsen accelerated approval. I count on you to not take away our hope.
Mom to Bobby Baird age 9