Sanfilippo syndrome

New marker found for Sanfilippo disease

Sanfilippo disease is a rare disorder caused by the failure of enzymes to break down specific kinds of complex carbohydrates, resulting in their accumulation…

UCLA scientists reveal how deadly pediatric disorder develops in brain

A deadly brain disorder in toddlers may find its first treatment in drugs for Alzheimer's disease. UCLA scientists have discovered how a form of the rare…

Researchers surprised by similar structures in Sanfilippo syndrome and Alzheimer's disease

Researchers seeking to understand the causes of a rare genetic lysosomal storage disease, Sanfilippo syndrome type B, were surprised to find protein…