Cure Sanfilippo Foundation Information

Cure Sanfilippo has funded 25 research grants around the world which has helped three clinical trials get underway, to date, treating children with Sanfilippo Syndrome. The Foundation creates and explores innovative and breakthrough research to ensure multiple promising paths are being explored in the effort to save children and better their quality of life. In addition to funding research, the Foundation has a focus on: 1) working with the U.S. Food and Drug Administration and biotechs regarding appropriate clinical trial endpoints and ensuring the caregiver voice is being incorporated every step of the way; 2) earlier diagnosis, diagnostic testing, and newborn screening; and 3) providing guidance and support to families for navigating the landscape of clinical trials, insurance and physician support.