My name is Betty Vertin. I live in Hastings, NE with my husband and five children. We have 12 year old daughter Lexi and 4 sons; they are Max, 8, Chance, 6, Rowen, 5, and Charlie, 3. In July of 2010, when I was just 10 weeks pregnant with our youngest, our son Max was diagnosed with Duchenne. In a matter of minutes a specialist told us what no parents ever want to hear: that our son would be wheelchair bound by 9, need our help with activities of daily living as a teenager, and would die before his 20th birthday. We woke up that morning with a beautiful healthy son and went to bed that night grieving the loss of a son that would come too soon. In the course of the next year, Rowen and Charlie were given the same fatal diagnosis. We believe, or at least hope the progression of the disease may not happen as quickly as the first doctor described to us; but we do know there is no cure and our sons will die because of the disease if there is no cure or at least better treatment options.
I am sharing with you because three of our four sons have Duchenne Muscular Dystrophy. I am sharing with you because I am scared that 3 of my 5 children could die without a cure or better treatments of Duchenne Muscular Dystrophy. My sons are young; but still face daily challenges because of Duchenne Muscular Dystrophy. Max is our oldest son, he is 8. Max's muscle strength is in a decline. There are things he could do three or four months ago that are becoming increasing more difficult for him. He is falling more often. Two weeks ago he fell and hurt the tendons around his knew. He limps and uses his medical stroller more now than ever before. We hope that as his tendons heal, his mobility will come back. However, because of Duchenne Muscular Dystrophy there is no guarantee that he will bounce back. Just yesterday he fell trying to play soccer, hit is head and has a concussion. He was falling like this three months ago. The hardest for me to swallow is that Max does not always finish putting a lego set together. That was unheard of a couple of months ago; Max would sit for hours to complete a set, but now he doesn't seem to have the stamina.
As a parent this is very hard to watch, no one should watch their children regress. Parents dream of watching their sons grow into men, watching their dreams come true. Duchenne and the lack of treatment threatens to steal this dream from us. It threatens not just the loss of one son, but three sons. It threatens to take half of my children from me. My husband and I feel we are proactive, we are doing everything within our power to save our children. We make financial sacrifice to take our children to the best doctor available (14 hours from our home), we follow a strict diet, we home stretch, we fund-raise to help finance research. In fact, Max participates in the Imaging DMD study and Ataluren trial in hopes that not only we help, but that he can have access to a drug that may save him and eventually his brothers.
Our experience with the Ataluren trial has been heart wrenching and I would like to share with you why. We screened Max for the study 6 months ago, before his decline, and he did not make it into the study because he walked too far during the 6 minute walk test. We re-screened this February and he made it in, and although we are happy he did, it was because his muscle strength had declined and he was no longer able to walk too far. If there had been no 6 minute walk test, Max could have had access to Ataluren before his decline and his strength could have been saved. We do not know if Max is receiving a placebo or the actual drug. However, based on the changes we have seen in his strength the last couple of months, I think it is very likely he is on the placebo. If there was no placebo, his change in muscle strength, his decline could be stopped. This is why I believe with all my heart, that every day that it takes to approve a drug, is a day my sons do not have. It is why I believe so strongly is a faster approach to approve the drugs that could help.
I want my sons to be saved. I want to watch all 5 of my children blossom into amazing adults and see all the things they accomplish. The possibility that I might see only two children live, should not be a possibility I have to consider as I think of the future. I have attached a picture of our family so that you can see those cute boys and know what we are fighting for and that you would join our fight to save them. Max, Rowen, and Charlie are the three we are holding.
A mother in love with her sons and not willing to let them go.