Brad and I formed this Foundation because of our four-year-old daughter, Kirby, the disease she was afflicted with, and our hope that Sanfilippo and its devastating progression would not manifest itself within her — our beautiful bundle of joy. The solution seemed simple. Raise funds to enable researchers to advance and expand their work to find a cure. We chose to fight this disease.
Now 19 years later, although Sanfilippo has taken its toll on Kirby, she continues to defy her affliction with seemingly endless resolve and still an occasional smile for us to cherish. A smile that brings us reassurance of her comfort and delight in her moments of happiness — a reminder of her amazing grace. She is our hero.
As her battle continues, our dream of a cure could come true because of the efforts of a team at Nationwide Children’s Hospital in Columbus, OH, that is ready to bring its gene therapy program to human clinical trial. Joining forces with other families and foundations, we hope to raise $1 million for testing required by the FDA to bring this work to fruition as early as December 2014.
The price to fund these studies might seem insurmountable. But all Brad and I have to do is remember what doctors first told us 19 years ago: Nothing can be done. Enjoy her while you have her.
Kirby's fortitude must not be in vain. No matter what the future holds for her, our mission is still for her. Because it is her story that is repeated in hundreds of families around the world, hundreds of stories shaped by one cure. And it has been your faithful belief in our mission that has allowed Kirby’s story to be written. It has been your unwavering support of the Foundation that has allowed it to issue grants totaling over $3.7 million and helped research to reach this level. It can be done.
From the early days when we chose to fight this disease and found only one researcher in the world working on Sanfilippo, to today when we face an exceptional opportunity, our mission to find a cure has never been more defined. We ask for your support in taking this final step to turn hope of a cure into a reality.
So raise a glass of Purple Lemonade and say cheers to these children!
Sue and Brad Wilson