I wanted to share an opportunity to help families who have children with Sanfilippo Syndrome fund gene therapy research. For years, families have been raising money to fund research - hosting lemonade stands, golf tournaments, 5k runs, galas, t-shirt sales. All of the hard work has paid off and we are on the cusp of gene therapy clinical trials moving forward at Nationwide Children's Hospital. They are currently running a Natural History Study for Sanfilippo and they hope to begin clinical trials within the next year or so. It will be a very small sample size, first for safety and efficacy. The hope is, if successful, there will be an effective treatment for Sanfilippo Syndrome.
A newly diagnosed family created the video above to raise awareness and money to help pay for the clinical trials. They have already raised over $500,000 in about 2 weeks. They have a goal of $1,000,000. Please consider donating to the Go Fund Me site: http://www.gofundme.com/ElizaONeill No amount is too small. And please share the video via social media.