Gala of Hope

Cure Spinal Muscular Atrophy

May 6, 2017 at Ritz-Carlton Laguna Niguel

by Nikki McIntosh

I still remember walking down the hospital hallway in a fog. After months and months of testing, the neurologist suspected that our then 18-month old son, Miles, might have a life-threatening disease. We got the call, the test was positive for Spinal muscular atrophy (SMA), a degenerative, neuromuscular disease that would slowly rob him of strength and lead to muscle atrophy. There was no treatment and no cure. So not only could our son not stand or walk, but it was expected that he would continue to grow weaker over time. The cruelty of the prognosis was heartbreaking and those first months after the diagnosis were the hardest time of our life. But then there was Miles. He was a little ray of light, living life with a smile on his face everyday despite his circumstances. We decided to take our cues from Miles and we chose to have hope. 

We sought out support and answers, which led us to the Cure SMA Foundation and the world of research towards a treatment and cure for SMA. We learned about best care practices for our son and we immersed ourselves into his care. 

At age two and a half, we started to see Miles regress. At around the same time, we learned of a clinical trial for a drug therapy that was showing promise in slowing the progression of the disease. Miles was enrolled into the trial and began receiving treatment. What we saw was nothing short of a miracle. Not only was the drug therapy stopping the progression of the disease, but it was also improving Miles’s strength.

On December 23, 2016, the FDA approved this drug therapy, Spinraza, and it became the first-ever treatment for SMA. For our family, the achievement of an approved treatment is an answered prayer and a hope realized. We have teamed up with the Cure SMA Foundation for the 3rd year, to chair The Cure SMA Gala of Hope on May 6th at the Ritz-Carlton Laguna Niguel. Being a part of the gala has been our way to help fight SMA and to bring hope to all the families like ours who are battling this disease. We now have an approved treatment and we’ll continue our quest all the way to a cure. We invite you to take part in this night of hope. For tickets or to donate, visit events.CureSMA.org/2017GalaofHope.