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.
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...
Cure Sanfilippo Foundation Chief Science Officer Cara O'Neill, MD, FAAP, and Board Member Valerie Byers, PhD, are among the contributors of a newly-published paper that identifies the most-important, early symptoms of Mucopolysaccharidosis III (MPS III) to assist...
The combined Sanfilippo Advocacy group submitted comments to the U.S. Food and Drug Administration's (FDA) on May 5, 2020, regarding its draft guidance on drug development for Sanfilippo Syndrome for review. These advocacy group is working closely with the FDA on the...
Cure Sanfilippo Foundation funds $380,000 to Phoenix Nest Inc. to support path to first-ever MPS IIIC Gene Therapy COLUMBIA, SC (May 15, 2020) — Cure Sanfilippo Foundation is thrilled to announce funding of $380,000 to the New York-based biotechnology company Phoenix...
On May 5, Cure Sanfilippo Foundation submitted the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome). The 39-page...
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.