A Breakthrough to a Cure Might be Here for Kirby and Other Children
After 16 years of supporting research to find a cure for Sanfilippo syndrome, The Foundation has received news of a promising breakthrough. A door has opened in our quest to find a cure for Kirby and other afflicted children. A 10-year-old gene therapy research program initiated by The Foundation is ready to move to human clinical trial.
There now is a timeline to a cure. The first step is to raise $1 million to fund preliminary studies required for FDA approval of human trial. Raising this money is The Children’s Medical Research Foundation’s fund-raising goal for 2011. Joining in this effort are other Sanfilippo families and foundations.
You can find details of this research which explains the work to be funded and the recent findings that offer so much hope for a lifesaving treatment. We ask for your contributions at this critical juncture.
If all goes well with the preliminary studies and FDA reviews, human clinical trial will follow directly. Additionally, The Foundation will continue to support the valuable and fruitful work being done in other areas of Sanfilippo research.
From the early days when we 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. And you have never failed to be there for Kirby and other afflicted children. We chose to fight this disease. Let’s take our biggest step together.
With gratitude,
Sue and Brad Wilson
