Research to find the first-ever treatment or cure for Sanfilippo Syndrome has come a long way in the years, but more work is needed.
This page provides access to information about the multiple therapeutic avenues being explored as a way to improve the quality of life for a child with Sanfilippo, the currently happening clinical trials regarding Sanfilippo, and what research the Foundation specifically has funded. And at the bottom of the page, you can access articles sharing information about progress and thought leadership among Sanfilippo research.
If you have any questions, especially if you are a parent of a child with Sanfilippo, please reach out to us at Contact@CureSanfilippoFoundation.org.
Therapeutic Avenues Being Investigated
What avenues for treating or curing Sanfilippo Syndrome are being explored by scientists?
Current Clinical Trials
See all the clinical trials underway for Sanfilippo Syndrome. We do our best to keep this list up to date, but we recommend you also check out ClinicalTrials.gov for the most current list possible.
Research Funded by Cure Sanfilippo
These are some of the most-recent scientific projects, including clinical trials, funded by Cure Sanfilippo Foundation. They range from basic research, to translational, and all the way to stage 1-2 clinical trials.
Our ability to fund these critical and promising research projects is because of the steadfast support of families impacted by Sanfilippo and the generous supporters who learn about this terrible disease and answer the call to help.
Identifying Disease Modifiers in Drosophila Models of Sanfilippo (MPS IIIA)
Grant Summary Institution: Clemson University, South Carolina, U.S. Primary Investigator: Trudy Mackay, PhD, Director, Clemson Center for Human Genetics and Self Family Endowed Chair of Human Genetics Duration: 24 months Start Date: January 2022 Type of Sanfilippo...
Discovery & validation of translational biomarkers for Sanfilippo childhood dementia
Page reviewed by: Dr. Cara O'Neill, FAAP and Jan Kaslin, PhD Page last updated: February 18, 2022 For web accessibility options: Click/tap the floating blue icon on the right. Grant Summary Institution: Monash University, Victoria, Australia Primary Investigator: Jan...
Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Sanfilippo
Grant Summary Institution: University of Naples Federico II, Naples, Italy Investigator: Luigi Michele Pavone, Associate Professor of Biochemistry, Department of Molecular Medicine and Medical Biotechnology, Medical School, University of Naples Federico II Duration:...
Exploring the role of heparan sulfate and dopamine as disease modifiers in Sanfilippo
Grant Summary Institution: Telethon Institute of Genetic Medicine (TIGEM), Pozzouli, Italy Principal Investigator: Elvira De Leonibus, PhD, of TIGEM and the Italian National Research Council Duration: 24 months Start Date: July 1, 2021 Type of Sanfilippo studied: A...
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...
Learn more about all of the research funded by the Foundation, as well the thought-leadership projects the Foundation supports, on the “What We Do” page.
Research & Science News
Find out the latest news and thought leadership regarding research on Sanfilippo Syndrome.