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 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 Sanfilippo Story
And the reality for every family living with Sanfilippo Syndrome.

We are writing the next chapter for Sanfilippo Syndrome. The chapter of the cure. Every project we architect or fund gets families closer to a cure in this lifetime.
$12.5 million raised since 2013

35+ research projects funded
100+ partner families globally


A cure can’t wait.
Every parent dreams about their child’s future and watching it unfold.
Sanfilippo Syndrome
takes that all away.
Sanfilippo Syndrome is a rare, terminal, neurodegenerative disease that causes children lose all the skills they’ve gained, suffer seizures and movement disorders, experience pain and suffering, and then die. All before their parents eyes.
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.
Has your child been diagnosed with Sanfilippo?
We are here to help.

With your support, we are writing the next chapter on Sanfilippo Syndrome.
The chapter of the cure.
Every project we architect or fund gets families closer to a cure in this lifetime.
$12.5 million raised since 2013
Medical & scientific expertise
35+ research projects funded
Multiple clinical trials funded
HOW YOU CAN HELP
YOUR SUPPORT HELPS FIND A CURE
HOW YOU CAN HELP
MEET THE
FAMILIES
OUR MISSION
The name says it all:
To cure Sanfilippo.
By advocating for and funding research directed towards a cure and treatment options.
FUNDRAISING GOAL
The Foundation’s focus in 2022: funding the most-promising research and elevating the voices of caregivers in pursuit of a cure for Sanfilippo syndrome.
Fundraising is critical fuel for these goals. Our goal is to raise $2,000,000 in 2022. We need your help to get there, especially in these turbulent times.
LATEST NEWS
Registration is open for ADVANCE 2022: Sanfilippo Community Conference
Jun 16, 2022
Registration is open for ADVANCE 2022: Sanfilippo Community Conference! A virtual conference on July 7-8, bringing together families and caregivers, scientists and researchers, clinicians and therapists, advocates, biotechs, and supporters. All to engage and advance...
Recent scientific articles relevant to Sanfilippo Syndrome: June 9, 2022, digest
Jun 9, 2022
Every week thousands of scientific articles on various topics are published. Here are some recent articles and abstracts that are relevant to understanding, managing, and/or treating Sanfilippo Syndrome (mucopolysaccharidosis III or MPS III)."The Inflammation in the...
Promising results from Lysogene MPS IIIA trial; novel PROVide study collecting observations
May 25, 2022
Lysogene announced May 18 that its AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPS IIIA was showing "promising efficacy data in young patients with persistent increase or stabilization." The clinical trial update was shared this...
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