Article

Mapping Sanfilippo Syndrome: A Multisystem Clinicopathological Autopsy

2026-03-26

Abstract excerpt

<h4>Background: </h4> /Objectives: Mucopolysaccharidosis type III (MPS III, Sanfilippo syndrome) is an autosomal recessive lysosomal storage disorder caused by deficiencies in enzymes required for heparan sulfate degradation. While primarily recognized for its devastating neurodegenerative course, the systemic extent of glycosaminoglycan (GAG) accumulation remains under-characterized. This study aims to provide a...

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Literature Corpus work: 3459ab30-a233-588b-8476-8308d4d930ce
DOI: 10.20944/preprints202603.2091.v1

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