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.

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...

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 regarding research on Sanfilippo Syndrome.