It Takes a Community
To achieve a cure, it takes families, researchers, clinicians, biotechs, and organizations working together. We are proud to be a part of this community and to help bring it together.
Get to Know the Community of Families.
Many Sanfilippo families work together with our Foundation to raise awareness and significant funding toward our annual fundraising goals.
We Keep Families Connected to Research and Fundraising.
We coordinate regular “Family Conference Calls” (typically every quarter) where we share the latest information, largely the progress of research and clinical trials, as well as current fundraising and awareness strategies and support.
We Are a Part of The Research & Advocate Community.
Cure Sanfilippo Foundation is proud to participate in multiple research groups and support the work of partner organizations.
Recent Collaborations
Sanfilippo researchers, leaders discuss data sharing to advance Sanfilippo Syndrome therapeutic development
Meeting summary now available to download Sanfilippo syndrome is an ultra-rare, neurometabolic disease which leads to severe neurodegeneration and multisystemic impacts for those affected. Individual heterogeneity, the protracted timespan of disease evolution across...
Testing of optimized gene therapy vector shows positive results in Sanfilippo type B mice
Newly-published research, led by Professor Coy Heldermon at the University of Florida, has described the use of an optimized gene therapy vector to treat a mouse model of Sanfilippo Syndrome Type B. Researchers used an AAV8 viral delivery vector that they had...
Sanfilippo parents unite on TikTok to raise $1M in 1 month for research for the deadly childhood disease
TikTok community donates more than $20,000 in 24 hours to support Sanfilippo research Several families of children with Sanfilippo Syndrome have been spreading awareness by sharing their journey with the disease and have amassed significant followings on TikTok over...
Sanfilippo research funding available from Foundation
Cure Sanfilippo Foundation now have three funding opportunities available for innovative research into Sanfilippo syndrome (MPS III). Cure Sanfilippo Foundation seeks to support research that fills critical gaps in current knowledge across basic science, clinical...
Replay of ADVANCE 2022, Sanfilippo conference, available on-demand
The virtual ADVANCE 2022, Sanfilippo Community Conference, July 7-8, 2022, brought together families and caregivers, scientists and researchers, clinicians and therapists, advocates, biotechs, and supporters. All to engage and advance the work to help children...
Collaborations & Partner Organizations
We Press For Research Findings To Be Shared.
The research we fund: We do our best to hold the researchers to standards of sharing their findings so others may build upon it, enabling a cure faster.