Every parent dreams about their kid’s future and watching it unfold.
Sanfilippo Syndrome takes all that away, replacing it with pain and suffering. The child experiences severe dementia and dies in their mid-teens. All before their parents eyes.
Cure Sanfilippo Foundation architects and funds cutting-edge research to accelerate the development of treatments and a cure for Sanfilippo Syndrome so parents can dream of their children’s futures again.
And the reality for every family living with Sanfilippo Syndrome.
Millions raised since 2013
15,000 children globally 50+ research projects funded
Engaging families around the world
4 clinical trials funded
Medical & scientific expertise
SANFILIPPO
MEET THE
FAMILIES
DONATE
Accelerating research to find a cure for children with Sanfilippo Syndrome
Every parent dreams about their child’s future and watching it unfold.
Sanfilippo Syndrome
takes that all away.
Cure Sanfilippo Foundation architects and funds cutting-edge research to accelerate discovery of a treatment or cure for Sanfilippo Syndrome, so parents can dream of their children’s futures again.
Help save children
VIDEO: One family’s story.
Every Sanfilippo family’s reality.
We are here to help.
With your support, we are writing the next chapter on Sanfilippo Syndrome.
The chapter of the cure for this childhood dementia.
Every project we architect or fund gets families closer to a cure in this lifetime.
-> $20 million raised since 2013
-> Medical & scientific expertise
-> 50+ research projects funded
-> Multiple clinical trials funded
DONATE
Find the
donation option
that fits you
FUNDRAISE
Easily create
and host your own
online fundraiser
TELL OTHERS
Make a difference
by telling others
about Sanfilippo
DONATE
Find the
donation option
that fits you
FUNDRAISE
Easily create
and host your own
online fundraiser
TELL OTHERS
Make a difference
by telling others
about Sanfilippo
FAMILIES
Cure Sanfilippo Foundation’s mission is to accelerate scientific development and access to a cure or therapeutic options for all affected by Sanfilippo Syndrome, to drive advocacy to improve care and outcomes, and to empower families with information, guidance, and support to navigate the journey.
ADVANCE 2026 conference recordings available
Recorded Sessions Available for On-Demand Replay The world of Sanfilippo Syndrome is changing fast. New research. New ideas. New possibilities. ADVANCE 2026 is your chance to get caught up on what’s happening and focus in on the topics you want to know more about. The...
Resource available to help families with out-of-pocket expenses due to Sanfilippo Syndrome Type A
The Assistance Fund (TAF) helps patients and families facing high medical out-of-pocket costs by providing financial assistance for copayments, coinsurance, deductibles, and other health-related expenses. Since its founding in 2009, TAF has helped over 260,000...
Patients Treated with Fayuvi Gene Therapy | MPS IIIA | Observational Study | Ultragenyx
Page last updated: October 5, 2026 Page reviewed by: Dr. Cara O'Neill, FAAPFor web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary The UX111-CL401 MPS IIIA Disease Monitoring Program is a prospective, multicenter, long term...
