One Family’s SANFILIPPO Story…

…but the reality for every family living with Sanfilippo Syndrome. Watch this video to learn why we exist, and why we need your help.

Learn More About Sanfilippo Syndrome
Watch the video about one family's fight to cure Sanfilippo Syndrome

Meet Our Sanfilippo Kids and Their Families

Cure Sanfilippo is powered by families. Our success is for and because of them. Hear their stories and you’ll understand why we fight so hard to do what we do.

Our Fundraising Goal

Our goal is to raise $2,000,000 in 2019. But we need your help to get there.

Learn Why We Raise Money
$1,230,000 raised
Fundraising goal for fight to cure Sanfilippo Syndrome
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Progressing research to cure Sanfilippo Syndrome

Curing Sanfilippo Syndrome is our ultimate goal, but we also want to influence the road to get there. With a 360-degree view, the Foundation funds extensive research into multiple avenues for a treatment/cure, as well as spearheads, supports, and funds initiatives that provide valuable, disease-specific and patient-preference information.

See What We’re Funding

Our Mission

To advocate for and fund research directed towards a cure and treatment options for patients with Sanfilippo Syndrome.

Learn More About What We Do

What’s Happening

Fun twist on ugly holiday sweater helps cure kids with Sanfilippo Syndrome

Fun twist on ugly holiday sweater helps cure kids with Sanfilippo Syndrome

Does an ugly holiday sweater help you get into the seasonal spirit?

How about one with unicorns!

Better yet, this sweatshirt also helps find a cure for Sanfilippo Syndrome.

So what are you waiting for? Grab what’s sure to be you and your kids’ favorite holiday shirts this year, and enjoy knowing that you’re also helping cure kids with Sanfilippo Syndrome.



The shirt’s artwork features prancing unicorns, evergreen trees, snowflakes, and the reminder that “All I Want For Christmas Is A Cure.”

Shirts are available in adult and children’s sizes. Order soon to ensure delivery in time for the holidays.

Proceed of the 2019 ugly holiday sweater benefit Cure Sanfilippo Foundation.

PLACE YOUR ORDER


*Special thanks to the Sarkar family for the idea, design, and modeling by big sister Sophia!

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Sanfilippo families & supporters lead Foundation to record-breaking 2019 Giving Tuesday

Sanfilippo families & supporters lead Foundation to record-breaking 2019 Giving Tuesday

The goal for 2019 Giving Tuesday, Dec. 3, was a big one: $100,000. Ambitious and hopeful, the Foundation collaborated with its partners families on a plan to far surpass the 2018 record of $43,000. At end of Giving Tuesday, more than $130,000 had been raised. 

Families of children with Sanfilippo Syndrome and their supporters hosted more than 50 individual fundraisers via Facebook in the week leading up to Giving Tuesday.

2019 Giving Tuesday main image

Donation gives children with Sanfilippo Syndrome the gift of more good days. And parents more quality time with their terminally-ill child. Plus … it may just save their lives.

A donation helps keep Carson playing baseball.
Keeps Liv laughing and smiling.
Keeps Spencer splashing in the pool.
Keeps Sadie singing her favorite songs.
Keeps Connor playing basketball and soccer.
Keeps Oliver giving hugs to everyone he meets.
Keeps Eliza playing in the backyard.
Keeps Will eating the pie he loves.

2019 Giving Tuesday e-mail array 1 2019 Giving Tuesday e-mail array 1

Donation helps fund promising research projects that could extend children’s quality of life, giving them and their families more good days together.

The Foundation provided content such as graphics, sample posts, videos, and funny takes on trending memes to support the individual fundraisers. 

Once again our Sanfilippo community came out strong. With all 53 Cure Sanfilippo Foundation fundraisers going on for Giving Tuesday, you have helped raise over $120,000!!, said Lisa Bittner, mom of Luke (MPS IIIA), posted on her 2019 Giving Tuesday Facebook fundraiser. “That is an incredible amount, and will make an impact on moving research forward, faster.”

We are always humbled by the love and support that our family receives not only today but everyday,” said Josephine Shamaly, mom of Ellie (MPS IIIB), on her 2019 Giving Tuesday Facebook fundraiser.

Several supporters waited until 8:00 a.m. ET on Giving Tuesday to compete for Facebook’s $7 million in matching funds. The competition for those matching funds is fierce and it will be weeks until the Foundation knows whether any of those 8:00 a.m. donations were able to snag a match.

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Drug Re-Purposing & Re-Positioning Studies Funded by Cure Sanfilippo Presented At International Conference

Drug Re-Purposing & Re-Positioning Studies Funded by Cure Sanfilippo Presented At International Conference

Last month, the research institute TIGEM presented preliminary results of two studies funded by Cure Sanfilippo Foundation at the Conference of Telethon Fundamental Associations in Italy.

Cure Sanfilippo partnered with TIGEM (Telethon Institute of Genetics and Medicine) on research projects to explore re-positioning and re-purposing FDA-approved compounds to determine if identify they can activate the clearance of pathologic lysosomal accumulation in MSD cellular models independently of the missing enzyme in each particular disease.

Posters were presented by TIGEM at the conference for both projects. 

Top of poster presentation at Telethon scientific meeting Oct 2019 about re-positioning FDA-approved compounds for MPS III


Bottom of poster presentation at Telethon scientific meeting Oct 2019 about re-positioning FDA-approved compounds for MPS III

The team developed a new high-content screening assay to determine lysosomal activity. They have identified similar primary “hit compound” in MSD and MPS IIIA. The same compound in the screening reduces cellular heparan sulfate GAGs in cell models for MSD and MPS IIIA.

Poster presentation at Telethon scientific meeting Oct 2019 about disease mechanisms and pharmacological targeting of MPS III

Read more about each specific research project:

Further work to determine efficacy and proper concentration doses and delivery is ongoing in animal models and efficacy.

Fellow Sanfilippo Advocate Katia Moletta (mother to Francesco, MPS IIIA) was able to be attend the scientific meeting and connect with the presenters from TIGEM.

Katia Moletta at Telethon scientific meeting Oct 2019   Katia Moletta and Sandro Montefusco of TIGEMat Telethon scientific meeting Oct 2019

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Foundation Update – October 2019

Foundation Update – October 2019

“This shouldn’t happen! Especially not to children.”

That’s what you thought when you first heard about Sanfilippo Syndrome. You put yourself in the shoes of these parents who were told that their children will suffer and die from Sanfilippo, and it broke your heart.
But you made the remarkable decision to something to help.
You donated to Cure Sanfilippo Foundation to find a treatment or cure. And your donation is an investment to create a different future.
You can see how your donation is helping cure Sanfilippo Syndrome.
More research is happening than ever before. More clinical trials than ever in the history of Sanfilippo. Including more than 20 projects and multiple clinical trials funded by Cure Sanfilippo in five years. And much more to come.
Check out the IMPACT REPORT.

Get a deeper look at what your generosity has already fueled. See the faces you are helping and the future you are creating. 






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None of this happens without you.

Thank you for your generous support. A cure is closer than ever because of you.

Deepest thanks,
Glenn O’Neill
Dad, Donor, President Cure Sanfilippo Foundation

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Cure Sanfilippo Foundation

501c3 non-profit
(Tax ID: 46-4322131)

curesff@gmail.com

P.O. Box 6901
Columbia, SC 29260

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