This poster was presented at the WORLDSymposium 2026 audience. Click on the poster to download a PDF of the poster.
Foundation Update
Update: Sanfilippo Type B Expanded Access opportunity and fundraising status
Just a month and a half ago, an opportunity arose to support the creation of a Sanfilippo Type B enzyme replacement drug for future use under an Expanded Access Program. Expanded access (EA) typically allows for broader access without the same restrictive inclusion...
Cure Sanfilippo Foundation is now accepting Letters of Interest (LOIs) for research program
Open call for proposals to ADVANCE Sanfilippo syndrome researchCure Sanfilippo Foundation is now accepting Letters of Interest (LOI) for its ADVANCE Sanfilippo Scientific & Clinical Research ProgramCure Sanfilippo Foundation supports research across basic and...
Foundation-funded translational research identifies dopaminergic drug that improves autism symptoms and helps restore dopamine-receptor activity in the Sanfilippo A mouse model
The research project further investigated the impact of altered heparan sulfate (HS) metabolism on brain development and its contribution to dementia-related protein aggregation Sanfilippo miceA two-year translational grant made in 2021 by Cure Sanfilippo Foundation,...
Foundation creates new position to elevate personalized, on-demand, Sanfilippo-specific support to families
Delivering the personalized information and support that you need, when you need itParents of children with Sanfilippo Syndrome face significantly more decisions on a daily basis than average parents. Not only are the decisions more in quantity, but also in...
Generous donors help raise $1,850,000 in 2023 for Sanfilippo research
Thanks to YOU, more than $1.85 million was donated to Cure Sanfilippo Foundation in 2023 to help children with Sanfilippo Syndrome. While this is a bit short of our $2 million annual goal, it's an incredible achievement in a difficult year for everyone, and we know...
10 years ago, we never could have imagined …
This month celebrates 10 years since we established Cure Sanfilippo Foundation, after our daughter Eliza was diagnosed with Sanfilippo Syndrome. Ten years ... it’s hard to believe. At this milestone, it felt natural to celebrate the progress you’ve enabled. And to...
April 2021 Research Update: New research in 2021 and promising projects already underway
Children should have the chance to grow up. Parents should never have to watch their children suffer and die. There needs to be a treatment or cure for Sanfilippo Syndrome. That’s why you support the Foundation's mission to help children with Sanfilippo. Critical...
Thankful for you; Looking back at 2020 and ahead into 2021
Our goal at the beginning of the year was to raise $2 million in 2020, to help further research and clinical trials for children with Sanfilippo Syndrome. But when the world shut down in March, it seemed so far away ... and honestly nearly impossible. But time and...