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.
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...
Facilitating and Enhancing CNS Entry of Systemically Delivered AAV Vectors for MPS IIIA, IIIB in Mice
Uniform gene delivery to the central nervous system (CNS) remains one of the most intractable challenges to the development of effective gene therapies for a wide range of neurological diseases, including MPS III (Sanfilippo Syndrome). While the brain microvasculature...
Cure Sanfilippo Co-Funds Research Into Treating Behavioral Symptoms of MPS III
Cure Sanfilippo Foundation has again collaborated with Sanfilippo Children's Foundation (Australia) to fund a new research project by Dr. Elvira De Leonibus at the Telethon Institute of Genetics and Medicine (TIGEM) in Italy. The research project aims to understand...
Cure Sanfilippo Funds CRISPR Gene Editing of Neural Stem Cells
Cure Sanfilippo Foundation is excited to grant funding to LA BioMed for the advancement of Dr. Michelina Iacovino’s work in the field of regenerative medicine for the treatment of Sanfilippo Syndrome (MPS III). This innovative work will utilize CRISPR gene editing to...
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.