Paying the Price

joseph and marissaYesterday was a historic day in the world of Duchenne. Sarepta Therapeutics announced that they’d finally received guidance from the FDA on moving the first Duchenne treatment through the approval process in an expedited manner. It may be coincidence, but it’s beautifully symbolic that, on the morning of Patriot’s Day, this news came from a company that is based in Boston. Patriot’s Day celebrates the start of the Revolutionary War, and it honors liberty and independence – two things we desperately want for our children battling Duchenne.

Since its inception in 1897, the Boston Marathon has always taken place on Patriot’s Day, and for so many, the interest in this year’s Marathon was on “one year later”: watching and gleaning bits of hope from stories of survivors and heroes. It was that way for me too, but my connection grew deeper and took on a significantly personal meaning a few months ago.

A young man in our community came to me and said he had qualified to run the Boston Marathon and wanted to do so on behalf of Team Joseph. Cory wanted to run and to raise awareness and research funds for all the kids who can’t run. It was remarkable to me that a high school senior, who is busy making graduation plans and looking forward to starting his freshman year at a Big Ten university next fall, would have the focus and the heart to use his platform to help others. That, in and of itself, is admirable, but there is another layer to the story.

What most people don’t know is Cory is a cancer survivor. At the age of 4, Cory was diagnosed with a rare form of cancer, and with the help of a protocol that included surgery, chemotherapy and radiation, Cory fought valiantly and won. And yesterday, just days after his 18th birthday, he ran to help us defeat Duchenne. As I watched the race live-streaming on my computer throughout the day, it struck me how significant it was that this young man could run a marathon. He was only with us, healthy and stronger than most, because someone fought for him. Someone developed a treatment. Someone believed in that treatment enough to shepherd it through the approval process. Someone paid the price for him to survive.

If you’ve ever benefitted from an antibiotic, that only happened because someone who came before you paid the price for you. If you’re a woman, you have the right to vote because someone who came before you paid the price. If you’re a person of color, you and I can go to the same restaurant and our children can attend school together because someone paid the price for us. Paying the price is never easy, but it is what creates a life of significance.

Paying the price is what The Race to Yes is about. To those of us who greet sleepless nights with endless questions and fears about the future for our children, the treatments will never come soon enough. But we have to begin somewhere, and that somewhere is here. That somewhere is right now.

I know yesterday was bittersweet for so many. There were worries and questions that reflected the fear of “what about the rest of us?” “What about those 87% of children who won’t be helped by eteplirsen?” I understand. I know those questions intimately. My own sweet son, Joseph, will not be helped by eteplirsen. He is missing exons 8 and 9. My friend Tracy Seckler understands. Her beautiful son Charley cannot be helped by eteplirsen.

When Tracy and I saw what a small group of parents were doing to fight for their boys who will benefit from eteplirsen, the only thing that made sense to us was to jump in and do our part. You see, eteplirsen is about so much more than exon 51.

Eteplirsen approval is about setting precedent. It’s a beginning. It’s about opening doors and blasting through walls when there aren’t any doors. It’s about digging deeper and finding courage and putting ourselves out there in the light of day where we can stand together and say, “We are not going away.”

The Race to Yes is about all potential treatments for all children with Duchenne. The Race to Yes is about unity and commitment and one singular focus – getting approval for treatments as quickly as possible. The Race to Yes is about staying the course and never letting up and doing the right thing. We have completed the first lap – a very significant pace-setting lap – but there is much to be done.

And so we’ll carry on: pushing forward and paying the price for all children with Duchenne – for all children in this generation and generations who aren’t even born yet.

I tuck my son into bed every night praying for a miracle. Tonight, I will celebrate the real potential of a treatment for that 13% of Duchenne children who will benefit from eteplirsen. At the same time, I will rub Joseph’s weakening legs, wondering when his answer will come. My dueling emotions confirm my belief that happiness and heartache can co-exist and travel together on this journey. I will lay my head on my pillow and recommit, promising Joseph that I will fight the fight until I have nothing left to give.

To the doubters, I say bring it on. I say thank you, for you make us more determined and fuel our fire.

To the supporters and the doers and the partners, I say thank you, for you are helping us stay the course, save lives and leave a legacy. You are helping us pave the way and pay the price.


Partners