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
Community’s vision for Rare Disease Center for Excellence at FDA
The Speeding Therapy Access Today (STAT ACT), H.R. 1730/S. 670, includes creation of a Rare Disease Center for Excellence within the U.S. Food and Drug Administration (FDA). The legislation was introduced March 2021 and is currently assigned to the House Committee on...
How sharing medical records can advance new treatments | AllStripes partnership
Sharing your child’s journey with Sanfilippo can advance new treatments Because Sanfilippo Syndrome is rare, researchers need more information directly from patients and their families to understand the condition. Your child’s progression, symptoms and health...
Advancing the Understanding of Special Education and Therapeutic Needs of Children with Neurodegenerative Disorders
The challenges of the pandemic shined a light on the critical role of special educators and rehabilitative therapists to protect the skills of children’ with neurodegenerative disorders. This academic publication and white paper provides teachers with a guide for...
Cure Sanfilippo lends support to multiple provisions of Cures 2.0 Act
The 21st Century Cures 2.0 Act legislation from U.S. Representatives Diana DeGette (D-Colorado) and Fred Upton (R-Michigan) could have a profound impact on the speed of therapy development and diagnostic access for patients with rare diseases. "Many organizations have...
Cure Sanfilippo agrees: The need to use surrogate biomarkers in an accelerated drug approval pathway for diseases like Sanfilippo
A powerful article from Dr. Emil D. Kakkis outlines the critical need for use of surrogate biomarkers in an accelerated drug approval pathway for diseases like Sanfilippo syndrome. In the article, "Aduhelm’s accelerated approval offers a promising roadmap for rare...
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.