News and information on issues relevant to you as a parent, caregiver, and advocate of a child with Sanfilippo Syndrome.
News From the Foundation
What Sanfilippo Families Need to Know: New Medicaid Work Requirements
Starting January 1, 2027, most states must require certain adult Medicaid enrollees to work, study, or volunteer 80 hours a month to keep their coverage. This comes from a new federal rule (CMS-2454-IFC) issued June 1, 2026. Many parents and caregivers of children...
Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB | MPS IIIB | Expanded Access | Spruce Bio
Page last updated: September 4, 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...
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...
“FDA’s inaction blocks progress for a cure” says Foundation’s O’Neill in USA Today op-ed
"Right now, 30 million Americans – half of them children – have rare diseases, 95% of which have no approved treatments. That’s why it’s so concerning that progress has been blocked by the recent rash of FDA denials of proposed treatments from drugmakers, despite the...
Neighbors, friends, and more make first Cure Clark 5K a huge success
More than 140 people turn out and help raise more than $10,000 to help children with Sanfilippo SyndromeThe community in the greater Lansing, Michigan, area spent the morning of August 23, 2025, helping a local family raise funds to cure Sanfilippo Syndrome. Japheth...
Foundation’s Dr. O’Neill publishes op-ed in The Hill, urging FDA to act
“The FDA must now ensure that its commitment to safety and efficacy is balanced with the urgency of patient need and the relentless progression of serious rare diseases. Action is needed now — before more life-saving therapies, and the patients who need them, fall...
Ultragenyx provides additional information regarding its UX111 program for Sanfilippo Type A
Ultragenyx Pharmaceutical Inc. has provided additional information regarding its UX111 gene therapy program for Sanfilippo Type A and the Complete Response Letter (CRL) it received from the U.S Food & Drug Administration (FDA) in July. They created a document...
Ambroxol Hydrochloride | MPS IIIA, IIIB, IIIC, IIID | Phase II-III | Lysosomal & Rare Disorders Research & Treatment Center
Page last updated: August 20, 2025 For web accessibility options: Click/tap the floating blue icon on the right.Trial Information Study title: An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol...
Action needed today: Senators need to hear from you
Good news and an urgent request to take action immediately … Two days ago, the Give Kids A Chance Act was unanimously passed by the House Energy and Commerce Committee and also by the full U.S. House of Representatives. This bill includes a 5-year renewal of the Rare...
Opportunity for Action: Ask Congress to Support Creating Hope Reauthorization Act/PRV
The Rare Disease Pediatric Review Voucher (PRV) is set to expire in late September. This would not be good for the rare disease community. The Foundation's thoughts on the PRV are: Pediatric Rare Disease Priority Review Voucher (PRV) program is a critical incentive to...
Further research confirms CLR01 reduces harmful clumping of abnormal proteins and brain inflammation, improves symptoms in Sanfilippo type A, B, and C mice
CLRO1 also enhances outcomes when combined with AAV gene therapyAssociate Professor Alessandro Fraldi and his team at CEINGE – Advanced Biotechnology in Naples, Italy, have completed their research project supported by a translational grant awarded in 2020 by Cure...
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC | MPS IIIC | Observational Study | Hospices Civils De Lyon
Page last updated: May 14, 2026 For web accessibility options: Click/tap the floating blue icon on the right.Study Summary In this multi-center, natural history study of subjects with Sanfilippo syndrome type C (MPS IIIC), patients will be monitored over a 2-year...
Breaking: FDA Agrees Heparan Sulfate is a Reasonable Surrogate Endpoint for UX111
MILESTONE BREAKING NEWS Ultragenyx Pharmaceutical Inc. announced just minutes ago that it held a successful meeting with the U.S. Food and Drug Administration (FDA), during which they reached agreement that cerebral spinal fluid (CSF) heparan sulfate (HS) is a...
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