We Are Working To Cure Sanfilippo Syndrome.
Our mission is simple:
To advocate for and fund research directed towards a cure and treatment options for patients with Sanfilippo Syndrome.
About the Foundation
Explore how the Foundation began, who leads it, the families that partner with it, and the impact it has had since it was founded.
Accelerate Research
Cure Sanfilippo conducts and funds promising research, projects, and clinical trials. Learn more about our research efforts.
Drive Collaboration
Together, a cure comes sooner. We collaborate with families, researchers, academic institutions, regulators, and bio-technology companies.
Increase Awareness
The more people who know about Sanfilippo, the more progress that happens to find a cure. So we help elevate awareness of Sanfilippo.
Raise Funds
We set ambitious annual fundraising goals in order to fund research for every kind of Sanfilippo and to dictate and accelerate the pace of research.
Support/Unite Families
Cure Sanfilippo connects families globally with the latest information on current research, as well as provides support for fundraising and caring for their children.
Cure Sanfilippo Foundation has come to represent a collection of Sanfilippo families (more than 80 globally and growing) and friends/supporters from across the country fighting to save our children under a single umbrella.
There’s HOPE for a cure, and that HOPE comes from the ACTION that is being taken from supporters like you, to further this mission to save children, and cure Sanfilippo.
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.
This is led by proactive families of children with Sanfilippo Syndrome and their supporters, who work tirelessly to change the fate and future for these children.
Donations may be tax-deductible.
Latest Foundation News
Denali announces plans to submit IND for ERT therapy DNL126 for Sanfilippo based on preclinical data
In a Feb. 22 press statement, Denali Therapeutics, based in San Francisco, California, announced it plans to seek Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) for its enzyme-replacement therapy (ERT) DNL126 designed for...
Packed days for Foundation leaders attending WORLDSymposium 2023, including presenting
Page reviewed by: Dr. Cara O'Neill, FAAPPage last updated: March 14, 2023For web accessibility options: Click/tap the floating blue icon on the right. Leaders of Cure Sanfilippo Foundation were once again busy during WORLDSymposium 2023. The six-day international...
WILLPower team raises nearly $50,000 in 2023 The Woodlands Marathon Charity Challenge
It's been 8 years since Valerie and Tim Byers learned that their young son had Sanfilippo Syndrome. Since then, they have ... Among their amazing annual fundraising efforts is leading the WILLPower marathon and fundraising team during the annual The Woodlands...
University of Manchester says gene therapy trial’s early results “show promise”
At WORLDSymposium 2023, Brian Bigger, Chair in Cell and Gene Therapy at University of Manchester, shared that its investigational gene therapy for Sanfilippo syndrome has "shown promising early results in a proof-of-concept study." The study was funded by Orchard...
2022 Annual Report: Look what you’ve done to help children with Sanfilippo
In late 2022, we shared with great excitement and appreciation that people's generosity was pushing Cure Sanfilippo Foundation well past the amount we thought possible to fundraise in a year. We are following up to share how this record-breaking year was made possible...
Upcoming Events
2023 Super Simon Says “Cure Sanfilippo Syndrome” Silent Auction | March 23-April 2, 2023
The family of Simon Croke is hosting the inaugural Super Simon Says "Cure Sanfilippo Syndrome" Silent Auction starting March 23, 2023, and ending with a Live Auction on April 2, 2023, benefiting Cure Sanfilippo Foundation atOrange Theory Fitness at the Triangle in...
2023 Super Eliza 5K | April 1, 2023 – Columbia, SC
The South Carolina College of Pharmacy PPAG group is hosting the 9th annual Super Eliza 5K Walk/Run to benefit Cure Sanfilippo Foundation and in honor of Eliza O'Neill on April 1, 2023.Sanfilippo Syndrome is a rapidly degenerative and fatal disease in children, much...
2023 Scotch Doubles Tournament | April 15, 2023 – Appleton, WI
The Mad Apple Burger & Billiard Co. in Appleton, Wisconsin, is hosting "2023 Scotch Doubles Tournament: A Fundraiser for Olivia" on April 15, 2023. This fundraiser benefits the fight to help Olivia Stoop, who lives in the community and has Sanfilippo Syndrome....
2023 Believe in G Golf Tournament | May 13, 2023 – Eureka, MO
Last year, the inaugural Believe in G Golf Tournament raised just over $𝟖𝟎,𝟎𝟎𝟎, benefiting the family and Cure Sanfilippo Foundation.Generous sponsors, donors, friends, family, volunteers, and our community made that $80,000 possible. The Wackers were overwhelmed by...
World Sanfilippo Awareness Day – Nov. 16, 2023 | Worldwide
World Sanfilippo Awareness Day is about spreading awareness and sparking conversations globally about Sanfilippo Syndrome, a disease few have heard of. This special day of Awareness is in honor of the children around the world living with Sanfilippo Syndrome today,...