As many of you know, Sanfilippo Syndrome is a rare disease. This means very little pharmaceutical money goes into research, given that our patient population is so small. (Not a large opportunity to make enough money to cover the research costs.) And federal funding for rare diseases has been drastically cut over the years. Therefore, it is up to families to come together to raise money.
There is some exciting research underway at Nationwide Children's Hospital. A few doctors there have been working for years on Sanfilippo Syndrome. They have done the mouse models, received approvals and are currently in the midst of a Natural History Study. Family run non-profits have funded them thus far. Moving into the human clinical trial phase is a huge undertaking and we need money to move it forward.
So Sanfilippo families are coming together with one goal: fund the clinical trial.
Please consider donating via our Crowd Rise page.