Sanfilippo Children's Foundation is dedicated to progressing clinical research into the effective treatment of Mucopolysaccharidosi s III, also known as MPSIII or Sanfilippo Syndrome.
Sanfilippo is a rare genetic condition causing fatal brain damage. It is referred to as a childhood disease because most patients never reach adulthood. There is currently no treatment and no cure.
Yet in the last few years, thanks to exceptional collaboration between patient groups, researchers and industry stakeholders, there has been an unprecedented level of cutting-edge medical research activity worldwide.
There is HOPE. Today there are a number of programs already in or about to enter clinical trial; these trials represent the only chance of treatment for children with Sanfilippo today.
You can help the Sanfilippo Children's Foundation to drive research and improve the quality of life for children with Sanfilippo and their families. Your support, donations and fundraising will allow us to finance critical research programs.
Our mission is to: "fund medical research so a cure can be found for children battling Sanfilippo today and for those born with it tomorrow".