The mission of Sanfilippo Initiative is to support research and clinical activity that aims to find and deliver cure for children suffering from Sanfilippo Syndrome
We are working to achieve our mission by:
  • Funding medical research relevant for finding a treatment for Sanfilippo with the aim to translate the innovative studies into clinical trials.
  • Supporting and enabling participation in clinical trials and promotion of compassionate use of promising therapies.
  • Providing diagnosed families with accurate and comprehensive information about latest available treatments.
  • Promoting awareness regarding in particular symptoms and potential therapeutic avenues among Pediatricians and general practitioners
  • Supporting constructive dialogue with health care specialists, pharma industry with the aim to allow broader access to potential treatment for patients with Sanfilippo and similar rare diseases
  • Raising awareness about Sanfilippo and similar rare diseases