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
Newborn screening pilot ScreenPlus that includes Sanfilippo Type A and B
When children with a rare disease are diagnosed at birth, they have the greatest opportunity to receive timely, approved treatments or participate in clinical trials that can provide them better quality of life and less pain and suffering. Early diagnosis and...
Foundation now accepting LOIs for Sanfilippo research funding
Cure Sanfilippo Foundation is now accepting Letters of Intent (LOI) 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 care, and...
2nd annual World Sanfilippo Awareness Day to be celebrated Nov. 16, 2020
People around the world are making a significant difference for children with Sanfilippo Syndrome. Each day, an effective, approved, and available treatment for Sanfilippo Syndrome is closer. Each year, World Sanfilippo Awareness Day is celebrated by this community...
Lack of appropriate education for children with significant special needs
Dr. Cara O'NeillChief Science Officer for Cure Sanfilippo Foundation The pandemic has Cure Sanfilippo Foundation embarking on some new advocacy work by Chief Science Officer Cara O'Neill. Many, many children with significant special needs, especially children with...
Cure Sanfilippo joins in calling for addition to Cures 2.0 legislation
Cure Sanfilippo Foundation is one of 46 patient advocacy organizations to sign-on to a letter asking U.S. Representatives Diana DeGette (D-Colorado) and Fred Upton (R-Michigan) to include H.R. 4144 - Ending the Diagnostic Odyssey Act in the Cures 2.0 legislation. The...
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.
We Encourage Families to Join the Global Registry
The more the patient population for Sanfilippo Syndrome is represented fully in a registry, the more interest there will be from industry to pursue and develop treatments. That’s why we fully inform our families about the existence of the Global MPS/ML Registry called Connect MPS.
If you are a new family or existing family with a child with Sanfilippo Syndrome, please make sure your child is registered.