What is Sanfilippo Syndrome?
Sanfilippo Syndrome (or MPSIII) is a genetic disease in children that is rapidly degenerative and terminal. Cure Sanfilippo Foundation’s mission statement is to advocate for and fund research directed towards a cure and treatment options for patients with Sanfilippo Syndrome. The SavingEliza.com campaign and funding all goes directly towards our non-profit goals.
About Eliza (Watch the 3 minute video below of the family story)
Eliza is a beautiful little girl, now almost 5 years old. Without something to stop this disease, her life expectancy is in the early teens or less. Today she runs, talks, laughs and plays. But she is beginning to show signs of disease progression. Rapidly in the next years she will lose all 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 as soon as possible. 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. Months, weeks, and days matter. We race to the finish line and the day children are treated. You are part of the cure.