Current Research on Sanfilippo

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.

Research Funded by Cure Sanfilippo

These are some of the most-recent scientific projects, including clinical trials, funded by Cure Sanfilippo Foundation.

Cure Sanfilippo Foundation Funds New Stem Cell Research

Cure Sanfilippo Foundation is pleased to support the work of Dr. Jan Nolta’s lab in collaboration with biotech partner Neuralstem. This project aims to develop an off-the-shelf neural stem cell therapy for patients with Sanfilippo Syndrome (MPS IIIA).As the Director...

Cure Sanfilippo Announces Support for Autophagy Research

Cure Sanfilippo Foundation is pleased to award funding support to Dr. Scott Selleck and the Pennsylvania State University (Penn State) to study the cellular process of autophagy as a central mechanism in MPS (mucopolysaccharidosis) disease progression in the central...

Re-positioning of FDA-Approved Compounds in MPS III Cell Models

Cure Sanfilippo Foundation is funding search to explore re-positioning FDA-approved compounds using cell-based, high-content screening in Sanfilippo Syndrome (MPS III) cell models. Awardee: Diego Medina, PhD- Telethon Institute of Genetics and Medicine (TIGEM)-...

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 regarding research on Sanfilippo Syndrome.