Zoey’s Miles for Muscles

Our daughter Zoey was born with a rare genetic disease called Spinal Muscular Atrophy (SMA). SMA is a condition in which gene SMN1 is missing, and causes muscle cells essentially to delete over time. It is the deadliest disease for infants if left untreated as eventually important muscle functions that support the cardiovascular system begin to fail. Zoey was diagnosed with the most severe form, Type 1.

Lucky for us, there are two treatments that are currently FDA approved. Spinraza is a drug administered by spinal tap that boosts the SMN1 backup gene, SMN2. This is the treatment Zoey receives and will receive every four months for life. Costs for this are $125,000 per shot. The second approved treatment is Zolgensma, which is gene therapy that aims to replace the missing SMN1 gene. This one time, one hour treatment delivered via IV is the most expensive drug in the world at roughly $2.1M (yes million) dollars. Because of these treatments, the outlook on SMA, while still incredibly deadly and serious, is brighter than ever.

CureSMA has been leading the way for advocacy and research for treatment. Without the work they have done, our little girl would currently be in hospice care with little to no options. The drug that gives her life, Spinraza, had research that started back in 2002 and was approved in 2016. Zolgensma was recently approved in 2019.

While we are blessed beyond measure to have 2 options, and while we stand on the shoulders of the parents who had zero options just a few years ago, it is not yet enough. These 2 options are only treatments, not cures. Whatever muscle ability a child has lost before treatment is lost - those cells do not come back. And while states drag their feet to create mandatory newborn screening for SMA, a cure is the real long-term solution.

We do not yet know what the physical limitations for Zoey will be. If she will run. If she will walk. And in this space of waiting, I have decided to use my ability to walk and run to try to bring awareness, as well as dollars, to the awesome work that CureSMA is doing.

I am asking for pledges, per mile, for calendar year 2020, to give to CureSMA, MDA, and other muscular dystrophy organizations. I plan on walking and running to raise awareness and funds for this cause. Please continue to join me, as many of you already have, in shouting #TeamZoey for all to hear. My goal is to get 500 miles during the year. There was no way for me to cap miles (to protect peoples wallets in case I became an ultra marathon runner by years end), but I will manually do so at 750 miles (in other words, if I somehow miraculously reach 750 miles by October, that will be the last piece of data I enter and the max amount pledged would be 750 * pledge amount ($.50, for example) = $375).

I will be collecting pledges to a personal bank account and then redistributing to the various organizations. As an accountant I am not worried about allocating funds, but I fully believe in transparency, and will be figuring out a way to show funds reaching the charitable causes as they should. Look for regular updates on the Facebook group “Zoey’s Miles for Muscles”.