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.
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...
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...
Research: Lysosomal storage disorders with altered heparan sulfate metabolism lead to dopamine-dependent autistic behaviors in Sanfilippo mouse models
A research project co-funded by Cure Sanfilippo Foundation and Sanfilippo Children's Foundation (Australia) recently published its findings, "Altered heparan sulfate metabolism during development triggers dopamine-dependent autistic-behaviours in models of lysosomal...
History is being made! Starting this week, newborns in the largest ever U.S. newborn screening study, ScreenPlus, are being screened for Sanfilippo syndrome Type B! Addition of Sanfilippo Type A will follow in the coming months! Why invest in Newborn Screening...
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.