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Sanfilippo syndrome, also known as mucopolysaccharidosis (MPS) type III, is a severe lysosomal storage condition that occurs when patients cannot break down long chains of sugar molecules. This causes devastating damage to the central nervous system. There are four subtypes of Sanfilippo syndrome (types A, B, C, and D). Early in childhood, patients experience severe developmental delay and regression. Over time, patients decline and many Sanfilippo patients do not survive past their teenage years. There is currently no cure for Sanfilippo syndrome.
The latest on our research
We are kicking off work with the Cure Sanfilippo Foundation on a new study. We want to make this study as strong as possible to accelerate more research, so we are currently recruiting more Sanfilippo families to join in this effort. We will share more details as the research progresses.