The Sanfilippo Children's Foundation is dedicated to progressing clinical research into the effective treatment of Mucopolysaccharidosis III, also known as MPSIII or Sanfilippo Syndrome.
Sanfilippo is a serious condition, delivering the most devastating outcome imaginable to patients of the disease and their families. But there is hope. There has been significant clinical progress in recent years and we believe that an effective treatment is within our reach.
Much of this progress has been due to the tireless efforts of foundations and biotech companies established by families and researchers with the specific objective to pioneer treatments, taking them from “bench to bed” within accelerated timeframes.