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)...
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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...
January 2026 Sanfilippo Speak: Oral Motor Function Therapy for Sanfilippo Syndrome
How can you proactively strengthen your child’s ability to eat and drink by mouth? What are the differences in traditional modalities of speech and communication therapy versus oral function, myofunctional, and feeding therapies? How can these modalities work for...
Update: Sanfilippo Type B Expanded Access opportunity and fundraising status
Just a month and a half ago, an opportunity arose to support the creation of a Sanfilippo Type B enzyme replacement drug for future use under an Expanded Access Program. Expanded access (EA) typically allows for broader access without the same restrictive inclusion...
November 2025 Sanfilippo Speak explores “total communication” with AAC
What is Alternative and Augmentative Communication (AAC)? What is “total communication”? How can AAC benefit both verbal and nonverbal communicators? How does the progression of Sanfilippo impact a person’s expressive communication skills and what can be done to help...