What is Happening In Sanfilippo Research?
Stay up to date on the latest information regarding scientific aspects of Sanfilippo Syndrome and progress to find a treatment or cure.
Recent News
Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB | MPS IIIB | Expanded Access | Spruce Bio
Page last updated: September 8, 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)...
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...
Sanfilippo Type B AAV9 gene therapy program is picked up by biotech
Sangrail Biologics, a clinical-stage gene therapy company focused on rare pediatric genetic diseases, announced the launch of the company on May 5, 2026, and announced its lead clinical asset, SNG-101 (formerly known as ABO-101 under Abeona Therapeutics), which is...
Leveraging clinical metadata in Sanfilippo syndrome: A scoping review, WORLDSymposium 2026 poster
This poster was presented at the WORLDSymposium 2026. Click on the poster to download a PDF of the poster.
Psychosocial burden in parents of individuals with Sanfilippo syndromes, WORLDSymposium 2026 poster
This poster was presented at the WORLDSymposium 2026 audience. Click on the poster to download a PDF of the poster.
Ultragenyx resubmits UX111 to FDA for accelerated approval of Sanfilippo Type A
Ultragenyx announced today that it has officially resubmitted its application to the U.S. FDA seeking accelerated approval of UX111, an AAV9 gene therapy for children living with Sanfilippo syndrome type A. With this resubmission, the FDA’s six month priority review...
Other Sources of News
ClinicalTrials.gov
Immediately see when clinical trials for Sanfilippo Syndrome are listed and detailed information.
Sanfilippo News
A site that aggregates news about Sanfilippo Syndrome into a single location.
RARE Cast – Global Genes’ Podcast
Hosted by award-winning journalist Daniel Levine, RARE Cast focuses on the intersection of rare disease with business, science, and policy in a weekly podcast.