Page last updated: August 17, 2026 For web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary This is an intermediate-size patient population Early Access Program (EAP) providing access to intracerebroventricular (ICV)...
Press Release
Erin Stoop, PharmD, mom of Olivia, joins Foundation Board of Directors
Erin Stoop, mom to 6-year-old Olivia (Sanfilippo Type B) is the newest addition to Cure Sanfilippo Foundation’s Board of Directors.Erin lives in Wisconsin with her husband Tyler, daughter Liv, and son Liam. Liv was diagnosed with Sanfilippo Syndrome Type B at age 2 in...
Natural History to Assess Disease in Patients With MPS IIIC (C-RARE) | MPS IIIC | Observational Study | UT Southwestern Children’s Medical Center
Page last updated: July 17, 2026 For web accessibility options: Click/tap the floating blue icon on the right.Study Summary This is a REMOTE prospective observational study of participants with Sanfilippo Syndrome Type IIIC (MPS IIIC). Patients’ functional abilities...
5 Things to Know | Sanfilippo Community Digest | July 10, 2026
“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things especially relevant to the Sanfilippo Syndrome community. We are going to send these regularly to keep you updated and put information and resources at your fingertips.1. Your...
Research platform for Sanfilippo syndrome, SanfilippoLINK, is now live and open for enrollment!
SanfilippoLINK is open to participants worldwide to advance understanding and treatments of all forms of Sanfilippo syndromeCure Sanfilippo Foundation is proud to announce that SanfilippoLINK, a global clinical research and patient registry platform dedicated to...
5 Things to Know | Sanfilippo Community Digest | June 12, 2026
“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things especially relevant to the Sanfilippo Syndrome community. We are going to send these regularly to keep you updated and put information and resources at your fingertips.1....
Spruce shares long-term data from weekly TA-ERT for Sanfilippo Type B at international conference
This month, Spruce Biosciences shared long-term data from its tralesinidase alfa enzyme replacement therapy (TA-ERT), an investigational treatment for Sanfilippo Syndrome type B (MPS IIIB), at the 18th International MPS & Related Lysosomal Diseases Symposium, June...
JCR to run clinical trial for JR-446 for Sanfilippo Type B in U.S. and Europe
Excited to see JCR Pharmaceuticals’ intravenous JR-446 enzyme replacement therapy (ERT) for Sanfilippo Syndrome Type B is now listed on ClinicalTrials.gov for a trial in the U.S., Germany, and United Kingdom. This is in addition to the trial JCR has been running in...
Mother’s op-ed urges FDA to stay committed to rare diseases patients
"The FDA’s New Leader Must Keep the Agency’s Promises to Rare Disease Patients"Elise Drake-Esposito, PhD, is a Research Project Coordinator for Cure Sanfilippo Foundation. She is also mother to Keira, her 16-year-old daughter who has Sanfilippo Syndrome, and she urges...