Today, May 15, is MPS (Mucopolysaccharidosis) Awareness Day. Sanfilippo Syndrome's official name is Mucopolysaccharidosis Type 3, or MPSIII. Sanfilippo is part of a group of lysosomal storage disorders known as mucopolysaccharidoses or MPS. The other related disorders...
Press Release
Family Friday 2023: The Esposito Family
Hear directly from the Esposito family – Dave, Elise, Keira, and Keaton!Awareness is a huge part of fighting Sanfilippo Syndrome. With awareness comes caring and understanding, which can lead to support and donations for research that can give children with Sanfilippo...
Family Friday 2023: The Laughlin Family
Hear directly from the Laughlin family – Shea, Erin, and Spencer!Awareness is a huge part of fighting Sanfilippo Syndrome. With awareness comes caring and understanding, which can lead to support and donations for research that can give children with Sanfilippo more...
U.S. patients: Denali’s natural history study still enrolling
Reminder: Denali Therapeutics is still enrolling patients with Sanfilippo Syndrome Type IIIA for its two-year natural history study. Denali Therapeutics is developing an enzyme replacement therapy for individuals with Sanfilippo type A. As part of their development...
Blood-based biomarkers for Sanfilippo syndrome progression and treatment
Grant Summary Institution: University of California, Los Angeles Primary Investigator: Gal Bitan, Ph.D./Professor of Neurology Duration: 24 months Start Date: September 2021 Type of Sanfilippo studied: Types A, B, C, and D Types of Sanfilippo that could benefit: Types...
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC (MPSIIIC) | Type IIIC | Observational Study | Hospices Civils De Lyon
Page last updated: April 24, 2023 For web accessibility options: Click/tap the floating blue icon on the right.Trial Information Hospices Civils De Lyon in France is conducting a single-site natural history study of patients with Sanfilippo Syndrome Type C (MPS IIIC...
Tips for Make-A-Wish trips to Disney World, from a Sanfilippo family
Many families choose a trip to Disney as their Make-A-Wish. While exciting, a Make-A-Wish trip to Disney can also be overwhelming for a family with a child with special needs to plan for. Thankfully, here are some tips for Make-A-Wish trips to Disney you can use to...
O’Neill joins other rare disease advocates for meeting with FDA Commissioner, Directors
Last week, Cure Sanfilippo Foundation Chief Science Officer and Co-Founder Cara O'Neill was privileged to be among a group of fellow ultra rare disease advocates meeting with the FDA Commissioner Robert Califf, MD, and Center Directors. The group presented the urgent...
Family Friday 2023: The Ulrich Family
Hear directly from the Ulrich family – Jericho, Christin, Elise, and Veda!Awareness is a huge part of fighting Sanfilippo Syndrome. With awareness comes caring and understanding, which can lead to support and donations for research that can give children with...