Stop SMA for Charlotte & Georgia

All 3 of our children were born with a disease called Spinal Muscular Atrophy, which works to rob them of their ability to move, breath and, eventually, live. There is a drug that can stop this disease. Health Canada has just approved it. But accessing it costs $354,000, and the process of our drug plan won’t be ready for another year. That’s a year that our girls can’t afford to wait. It would be a year of vulnerability and loss. 


But with your help, we believe we can turn it into a year of miracles!

Let me tell you the rest of our story.

Spinal Muscular Atrophy (SMA) has become our daily life. It’s the genetic condition most likely to kill children under two years of age, and all 3 of our children have it. It’s a progressive lower motor neuron disease, that is loosely paralelled to adult ALS. But there are significant differences: most notably, SMA never affects cognition, so our kids are as bright and keen as any other kids their age.

But the physical decline has been steep. Georgia just turned 6.


At 14 months, she took her first 3 steps. She has never walked since then. After those first steps, she lost any strength to walk, then strength to pull up to stand, and then the strength to crawl. She can barely get from a lying position to a sitting position. Georgia is our mature little scholar! But she is completely dependant on us for dressing, toileting, and getting her in her wheelchair.But those things aren’t the worst. She is at an extreme risk of respiratory failure. Every single simple cold/viral infection is a threat. At least once a year these infections strike hard enough to send her to the hospital. As a father, I weep and get nauseous just thinking about this. I don’t want to think about what the next year could hold for her.

Charlotte is 4, and for her the risks are different. She has type 3 SMA, and she is our tireless ball of enthusiasm! We are so grateful she has maintained the ability to walk. But we can see the weakness advancing.


First she stopped running, and then walking up stairs. Now, she collapses onto the ground regularly, because of her weakness, and it’s hard for her get to a standing position. Given this trajectory, it’s almost certain she’ll lose the ability to walk at all by the end of another year.  It’s scary. 

But the amazing thing is that it doesn’t have to be that kind of year for our girls!

For over two decades the gene responsible for SMA has been known but there has not been a single available treatment. This summer the first drug in history to specifically treat SMA was approvd by Health Canada! It is called Spinraza (Nusinersen). It can completely halt SMA in its path and even lead to some GAIN in muscle strength! 

See our family on Global News

We know first hand: our one-year-old son Thomas is in an open label trial at Johns Hopkins University, and he has already received 6 doses of Spinraza intrathecally. He is symptom free! And he is showing no signs of slowing down. There is true hope that he will remain symptom free for his entire life! There is no such trial available for Georgia and Charlotte.  

This drug truly is a miracle, and we need all of our kids on it as soon as possible.

Though it’s approved by Health Canada, Spinraza won’t be available on our drug plans for another year. It costs $354,000 to provide a year’s supply for Georgia and Charlotte, 3 vials each. The manufacturer, Biogen, will sell it directly to families willing to pay for it.

This treatment WILL stop Georgia’s respiratory deterioration, keep her hands strong, and prevent future problems in her spine that would require surgery. It WILL save Charlotte from losing the ability to walk. And we don’t even know what gains it might provide. Instead of a year of atrophy it can become a year of miracles! But we need your help. We do everything we can for our girls but we can’t do this alone. We need to raise $354,000. 


Please join us on our journey and help to make this a year of miracles for Charlotte and Georgia!