The Assistance Fund (TAF) helps patients and families facing high medical out-of-pocket costs by providing financial assistance for copayments, coinsurance, deductibles, and other health-related expenses. Since its founding in 2009, TAF has helped over 260,000...
Press Release
Patients Treated with Fayuvi Gene Therapy | MPS IIIA | Observational Study | Ultragenyx
Page last updated: October 5, 2026 Page reviewed by: Dr. Cara O'Neill, FAAPFor web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary The UX111-CL401 MPS IIIA Disease Monitoring Program is a prospective, multicenter, long term...
Patients Treated with UX-111 (Clinical Trial) Gene Therapy | MPS IIIA | Follow-Up Study | Ultragenyx
Page last updated: October 5, 2026 Page reviewed by: Dr. Cara O'Neill, FAAPFor web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary This is a multicenter, long-term follow-up study of patients with MPS IIIA who have...
Mohammed Harris, PhD, joins Foundation staff to continue advancement of critical Sanfilippo research programs and communications
There has been an exponential increase in the pace of investigation and investment into Sanfilippo Syndrome over recent years, thanks to the decades of work by families, researchers, and clinicians to increase the understanding of this terminal disease and explore...
NGT-104 AAV9 Gene Therapy | MPS IIIB | Phase I-II | NeuroGT
Page last updated: October 5, 2026 Page reviewed by: Dr. Cara O'Neill, FAAPFor web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary The goal of this clinical trial is to learn if one infusion of NGT-104 (rAAV9-CMV-hNAGLUop)...
FDA approves first-ever treatment for Sanfilippo Syndrome Type A, FAYUVI™
On September 17, 2026, the U.S. Food and Drug Administration (FDA) granted standard full approval of FAYUVI™ (rebisufligene etisparvovec-hopf), also known as UX111, for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo...
What happens when families, researchers, clinicians, and biotechs get the chance to learn from each other?
Find out at ADVANCE 2026, Sept. 22 & 23, the virtual Sanfilippo community conference, hosted by Cure Sanfilippo Foundation!A research finding can spark a new question from a parent. A family's experience can offer a researcher or clinician a perspective they...
First patient dosed in JCR/MEDIPAL’s JR-446 ERT global clinical trial for Sanfilippo Type B
JCR and MEDIPAL Holdings announced on September 11, 2026, that the first patient was dozed in its global Phase I/II clinical trial of JR-446 for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB). JR-446 is a blood-brain barrier-penetrating...
2026 Hands-on Workshop: “Does my insurance cover this?”
“Does my insurance cover this?” This is a common question Sanfilippo families face. If combing through your insurance policy feels like reading a foreign language, Cure Sanfilippo Foundation is here to help! In this small group, hands-on workshop, we dive into the...