What is Sanfilippo Syndrome?
Sanfilippo Syndrome (or MPSIII) is a terminal genetic disease. It is degenerative and Eliza’s life expectancy is now in the early teens or less. Today she runs, talks, laughs and plays like any child her age (just turned 4). Rapidly in the next years she will lose the capacity for speech, walking, swallowing, and ultimately the loss of vital organ functions. Most children have lost their ability to speak by the time they are 6 and will have severe brain damage. This will happen with 100% certainty without treatment. Currently there is no cure, nor is there any treatment to slow the condition. It is a parent’s worst nightmare and an unfair sentence for any innocent child.
Gene Therapy at Nationwide Children’s Hospital in Ohio have stopped Sanfilippo in animal tests. Funding is critical and urgently needed to move the clinical trial to the human stage and keep it on schedule for late 2014. Every dollar counts in the goal to save these children. Time is everything to a Sanfilippo Parent in an effort to treat their children before significant symptoms and disease progression. We all race to the finish line and the day our children are treated. To be part of stopping this deadly childhood disease in 2014 is something very special. You are part of the cure.