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.
And the reality for every family living with Sanfilippo Syndrome.
$20 million raised since 2013
15,000 children globally 50+ research projects funded
120+ partner families globally
3 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
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
To cure Sanfilippo.
By advocating for and funding research directed towards a cure and treatment options.
The Foundation’s focus in 2023: 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 $2,500,000 in 2023. We need your help to get there. Every single donation makes a difference.
JR-441 Enzyme Replacement | Type IIIA | Phase I-II | JCR
Page last updated: October 23, 2023 For web accessibility options: Click/tap the floating blue icon on the right. Trial Information Phase I/​II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA A Phase I/ II, open-label, randomized, 2-arm study,...
$8 million awarded to study brain cell death in fatal pediatric diseases, including Sanfilippo
Washington University School of Medicine (St. Louis, MO) has received nearly $8 million from the National Institute of Neurological Disorders and Stroke of the National Institutes of Health (NIH) to help determine the root causes of brain cell death in fatal pediatric...
Families & friends fighting to ensure Sanfilippo research continues
We are so thankful for the many families who continue hosting events and fundraisers to support critical research -- especially early, novel and translational research -- that the Foundation funds, in addition to clinical trials. Here are several fundraisers that have...
