Current Research on Sanfilippo

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

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 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. 

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...

Re-Purposing FDA-Approved Inflammation Drug for Sanfilippo

Cure Sanfilippo Foundation is sponsoring an “Open-label Study of Anakinra in MPS III” clinical trial in collaboration with The Lundquist Institute (formerly LABiomed), and Sobi (Swedish Oprhan Biovitrium AB). This study is an open-label, single-arm, no placebo or...

Neural Stem Cells for Sanfilippo Syndrome

Cure Sanfilippo Foundation has committed funding for a multi-year grant to Karl Johe, PhD, of ReMotor Therapeutics, Inc., to explore the therapeutic benefit of transplanting human neural stem cells engineered to overexpress and secrete SGSH into the brain of patients...

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.