Sanfilippo Is a Rare Disease, But There Is Greater Awareness Than Ever.
The compassion of people is truly amazing. In five years, more than $8 million has been donated to 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 40,000 strangers have stepped up to help find a cure for Sanfilippo Syndrome.
We work to keep elevating awareness by constantly telling the story. For one Sanfilippo family’s story is the reality for all of us.
Thought-Leadership Projects
Curing Sanfilippo is our ultimate goal, but your support also allows us to play a critical roll in influencing the road that leads there. Learn more about the Foundation’s thought-leadership projects.
Collaborations
Curing Sanfilippo is our ultimate goal, but your support also allows us to play a critical roll in influencing the road that leads there. Learn more about the Foundation’s collaborative mindset.
Sanfilippo Syndrome In The News
2026 Hands-on Workshop: “Does my insurance cover this?”
“Does my insurance cover this?” This is a common question Sanfilippo families face. If combing through your insurance policy feels like reading a foreign language, Cure Sanfilippo Foundation is here to help! In this small group, hands-on workshop, we dive into the...
5 Things to Know | Sanfilippo Community Digest | Aug. 24, 2026
“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things especially relevant to the Sanfilippo Syndrome community. We are going to send these regularly to keep you updated and put information and resources at your fingertips.1. Latest...
What Sanfilippo Families Need to Know: New Medicaid Work Requirements
Starting January 1, 2027, most states must require certain adult Medicaid enrollees to work, study, or volunteer 80 hours a month to keep their coverage. This comes from a new federal rule (CMS-2454-IFC) issued June 1, 2026. Many parents and caregivers of children...
Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB | MPS IIIB | Expanded Access | Spruce Bio
Page last updated: September 4, 2026 For web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary This is an intermediate-size patient population Early Access Program (EAP) providing access to intracerebroventricular (ICV)...
Erin Stoop, PharmD, mom of Olivia, joins Foundation Board of Directors
Erin Stoop, mom to 6-year-old Olivia (Sanfilippo Type B) is the newest addition to Cure Sanfilippo Foundation’s Board of Directors.Erin lives in Wisconsin with her husband Tyler, daughter Liv, and son Liam. Liv was diagnosed with Sanfilippo Syndrome Type B at age 2 in...
Natural History to Assess Disease in Patients With MPS IIIC (C-RARE) | MPS IIIC | Observational Study | UT Southwestern Children’s Medical Center
Page last updated: July 17, 2026 For web accessibility options: Click/tap the floating blue icon on the right.Study Summary This is a REMOTE prospective observational study of participants with Sanfilippo Syndrome Type IIIC (MPS IIIC). Patients’ functional abilities...