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
Family Friday 2023: The Stoop Family
Hear directly from the Stoop family – Tyler, Erin, Olivia, and Liam!Awareness is a huge part of fighting Sanfilippo Syndrome. With awareness comes caring and understanding, which can lead to support and donations for research that can give children with Sanfilippo...
Recognizing MPS Awareness Day 2023
Today, May 15, is MPS (Mucopolysaccharidosis) Awareness Day. Sanfilippo Syndrome's official name is Mucopolysaccharidosis Type 3, or MPSIII. Sanfilippo is part of a group of lysosomal storage disorders known as mucopolysaccharidoses or MPS. The other related disorders...
Family Friday 2023: The Esposito Family
Hear directly from the Esposito family – Dave, Elise, Keira, and Keaton!Awareness is a huge part of fighting Sanfilippo Syndrome. With awareness comes caring and understanding, which can lead to support and donations for research that can give children with Sanfilippo...
Family Friday 2023: The Laughlin Family
Hear directly from the Laughlin family – Shea, Erin, and Spencer!Awareness is a huge part of fighting Sanfilippo Syndrome. With awareness comes caring and understanding, which can lead to support and donations for research that can give children with Sanfilippo more...
U.S. patients: Denali’s natural history study still enrolling
Reminder: Denali Therapeutics is still enrolling patients with Sanfilippo Syndrome Type IIIA for its two-year natural history study. Denali Therapeutics is developing an enzyme replacement therapy for individuals with Sanfilippo type A. As part of their development...
Blood-based biomarkers for Sanfilippo syndrome progression and treatment
Grant Summary Institution: University of California, Los Angeles Primary Investigator: Gal Bitan, Ph.D./Professor of Neurology Duration: 24 months Start Date: September 2021 Type of Sanfilippo studied: Types A, B, C, and D Types of Sanfilippo that could benefit: Types...