I have two sons with DMD. Elliott is now 10 and Henry is 7. You feel helpless as a parent as you watch them get older and lose ability and you can do nothing to stop it. We are blessed that both are functioning fairly independently, but we see decline in function as each year passes.
It is heartbreaking to see your child struggle with things we do without thinking- like unscrewing the cap off of a tube of toothpaste, sticking a straw into a juice box or clearing just one step. I would do anything as a parent to stop or slow down progression and to help them maintain some form of independence for as long as they can. With that in mind, we were thrilled that my older son was able to participate in a clinical trial starting back in 2008. Five years later, still no FDA approval, but we continue to participate, because we believe it is helping him, no adverse effects have been reported and to be honest, it's better than doing nothing.
I want to be able to tell my sons that I did everything possible. At the end of the day, I feel that is my job as their mother. Initially my younger son was not able to participate because he did not meet the minimum age requirement. That was a difficult decision to make, but we were hoping that in a few years the drug would be close to FDA approval. Things did not go as planned and five years later, our only hope is that our younger son will be able to participate in a confirmatory study for the drug. He may be taking placebo or drug, we won't know. I feel this is cruel and unusual punishment, especially given the time we have already invested in this clinical trial. I am not alone in this difficult position.
I would like to see the FDA consider sibling arms to these trials or work with drug companies to determine ways to give access to siblings who do not meet eligibility requirements. All of them deserve a chance.