We’re working to cure Sanfilippo Syndrome
When our daughter Eliza was diagnosed with Sanfilippo Syndrome at the age of four, we decided we had to do everything possible to find a cure. How could we live with ourselves if we didn’t? How could we face our son and Eliza’s brother if we didn’t? So, we created the Cure Sanfilippo Foundation in order to pursue every avenue to change the fate of every Sanfilippo children, not just ours. The Foundation started in 2014 as our “family” foundation, but we’ve come to represent a collection of Sanfilippo families and friends from across the country fighting to save our children under a single umbrella. There’s HOPE for a cure, and we’re making sure there’s plenty of ACTION to make it a reality.
– Glenn and Cara O’Neill, parents of Eliza
Our mission is simple:
To advocate for and fund research directed towards a cure and treatment options for patients with Sanfilippo Syndrome.
Cure Sanfilippo Foundation is a 501(C)(3) not-for-profit organization.
All net funding goes to the urgent mission to advance treatment options to treat children with Sanfilippo. Our volunteers are in the hundreds who support our Foundation, our events, and our campaigns. Donations are tax-deductible.
Please contact us:
Whether you are the family of a child with Sanfilippo, a researcher looking for funding, or simply someone who wants to get involved and help raise awareness, we would love to hear from you.
Mail: PO Box 6901, Columbia, SC 29260
Learn More About What We Do:
- We Raise Money & Fund Research
- We Drive Collaboration
- We Increase Awareness
- We Support and Unite Sanfilippo Families
- Who Leads the Foundation
We Raise Money & Fund Research
Our efforts are global and far reaching as our goal is to end a disease. When the disease first starts showing around age 3, we parents are just beginning to see our child’s personality and potential….and then it is all taken away. Today, nothing exists to stop this, but real HOPE is on the horizon. Breakthrough science has discovered treatments and a hopeful cure which have proven in research models to slow or stop Sanfilippo. We fund this most promising research to bring about more clinical trial options…a life line for these kids. But we need your help in raising these funds.
Each year, we set huge fundraising goals
Many might wonder, “Why so much?” Our Foundation has a vision of not just funding research for a cure for every kind of Sanfilippo as it becomes available, but instead being so influential that we’re dictating the pace of research. That when promising ideas and research emerge, we’re able to make lab capacity, fund facilities that quickly take ideas forward to clinical trials.
How are those funds spent?
Every single dollar matters, and with our organization running on all volunteers from our Board to our brilliant volunteer Scientific Advisors, every net dollar goes to this urgent research. That might be drug production for more children to be treated, clinical trial resources, breaking research in enzyme replacement, non-embryonic stem cells, chaperone therapies, or tackling the antibody suppression issue in gene Therapy. Time is not on the children’s side, but HOPE is. your compassion is making the difference and our deepest thanks for your support for this life saving mission.
We fund a variety of projects
While there are many Sanfilippo foundations and families funding research around the world, the following are projects being funded by the Cure Sanfilippo Foundation:
- Funded pre-clinical Steps and Funding drug Production for the planned gene Therapy Clinical Trial (UPDATE: Type A clinical trial approved by FDA to proceed).
- Funded critical positions at Nationwide Children’s Hospital working on the preparations for the gene Therapy Clinical Trials.
- Funding novel Enzyme replacement study, Mesenchymal Stem Cell (non-embryonic) study, & testing for Chaperone Therapy.
- Funding needs for Neutralizing Antibodies, additional clinical Trials for more children.
Apply for foundation funding
If you’re a clinical researcher and are interested in funding from the Cure Sanfilippo Foundation, please email us and we can begin a conversation.
We Drive Collaboration
When you receive the diagnosis of Sanfilippo, you immediately want answers. What does this mean? Where’s the research? Who can I talk to?
We’re a community, here to share information, the latest research, how to fundraise and spread awareness.
- Many Sanfilippo Families working together with our Foundation raising awareness and significant funding toward our $2M goal in 2016.
- Grassroots Family-run Fundraising efforts: golf Outings, Auction Parties, Fishing Tournament, 60+ national coordinated yard/garage sales on one day, etc…
- Cure Sanfilippo Foundation holds monthly calls for Sanfilippo families to share information, discuss progress on research & clinical trials, gather community feedback, and collaborate on ideas.
To see how individual families are raising awareness through social media, visit Meet the Families.
We keep families connected to research and fundraising.
We coordinate regular “Family Conference Calls” (typically every quarter) where we share the latest information, largely the progress of research and clinical trials, as well as current fundraising and awareness strategies.
We drive a network of sharing.
We coordinate quarterly “Foundation Conference Calls” with Foundations around the world. We’re constantly leading the charge in pursuit of the research and moving it toward clinical trials. Information is empowering, so we’re always looking for ways to share everything.
Demanding shared research findings.
The research we fund: We do our best to hold the researchers to standards of sharing their findings so others may build upon it, enabling a cure faster. We don’t believe in proprietary research; we fund a community.
We Increase Awareness
The compassion of people has amazed us. In three years, more than $3 million has been raised by the Foundation. All of it coming from individuals who heard the stories of Sanfilippo families and made the selfless choice to help our kids have a chance at life. More than 25,000 strangers have stepped up in just three years to support the Cure Sanfilippo Foundation, and it’s all because of the awareness raised by Sanfilippo families.
While a rare disease, we refuse for that to mean the diseases stealing our children’s future is ignored. We work constantly, telling our story (for one Sanfilippo family’s story is the reality for all of us).
We Support and Unite Families
We pool our resources. Together, we can accomplish amazing things. We welcome all Sanfilippo families to join our Foundation’s fight to cure this disease. We are open to you if you’re interested in more information about Sanfilippo, more details about where the most promising research stands, best practices we’ve experienced, to participate in fundraisers, or even formally roll up under our Foundation. Just contact us and join the family.
Who Leads the Foundation
- Glenn O’Neill
- Cara O’Neill MD FAAP
- Rachael Best
- Daniel Fraley
- Shelby Leonardi
- Faith McAngus
- Katie Walton
- Valerie Byers
Scientific and Physician Advisiors
- Tim Wood, PhD
- Jeffrey D. Esko, PhD
- Jan Nolta, PhD
- Debbie Greenhouse, MD FAAP
- Gustavo Maegawa, MD, PhD, FABMG