Sanfilippo Syndrome Foundations

Cure Sanfilippo Foundation is a leading non-profit organization dedicated to accelerating the discovery of a cure or effective treatment for Sanfilippo Syndrome (Mucopolysaccharidosis type III), a rare and terminal neurodegenerative disease often described as childhood dementia. Founded in 2013 by Glenn and Cara O'Neill following their daughter Eliza's diagnosis, the foundation has raised over $20 million to fund more than 50 research projects and multiple clinical trials globally. The foundation's strategic investments focus on high-impact therapeutic modalities, including gene therapies, enzyme replacement therapies, and small molecule treatments, while also driving advocacy to improve clinical care and patient outcomes. By partnering with academic institutions and biotechnology companies, Cure Sanfilippo Foundation plays a pivotal role in advancing the clinical pipeline for all four types of Sanfilippo Syndrome.