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...
Scientific News
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...
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...
“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...
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...