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.
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...
Strengthening the rationale for the use of the “molecular tweezer” CLR01 in the treatment of Sanfilippo
Cure Sanfilippo Foundation has co-funded a research grant with Sanfilippo Children's Foundation (Australia) and H.A.N.D.S consortium, an international network of Type C families and groups. This grant is to Associate Professor Alessandro Fraldi at CEINGE - Advanced...
Investigating NDST1 inhibitors as target for substrate reduction therapy for Sanfilippo
Identifying a novel class of substrate reduction therapy drugs for the mucopolysaccharidoses that inhibit N-deacetylase/N-sulfo-transferase (NDST) Cure Sanfilippo Foundation has co-funded a one-year research project with Sanfilippo Children’s Foundation (Australia) to...
Exploring anti-fungal immunity and the airway microbiome in Sanfilippo syndrome (MPS III)
Cure Sanfilippo Foundation is funding a two-year project by Neta Shlezinger, PhD, at the Koret School of Veterinary Medicine in Israel, in collaboration with Dr. Annick Raas-Rothschild, Associate Professor and Director of Rare Diseases Institute at Sheba...
Creation of plasmid and efficacy study for MPS IIIC clinical trial
Cure Sanfilippo Foundation has funded New York-based Phoenix Nest, Inc. The funding supports creation of plasmid and the required in-vivo efficacy study in the Sanfilippo Type C (MPS IIIC) mouse model. These and other steps will enable the project to be evaluated by...
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.