Silent Auction - Coming 2021
The families of children with Sanfilippo are a community who fight together. Hear their stories & how you can help now.
Sanfilippo Syndrome is a fatal progressive disease that primarily affects the brain of children born with this disease. A genetic defect passed on from each parent results in missing or poorly functioning enzymes needed for cells to work normally. Without these enzymes, cells are unable to break down and recycle cellular waste. Over time, this waste builds up causing cells act abnormally and then die.