Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo Disease

The mucopolysaccharidoses (MPS) are a group of diseases caused by the lysosomal accumulation of glycosaminoglycans, due to genetic deficiencies of enzymes involved in their degradation. MPS III or Sanfilippo disease, in particular, is characterized by early-onset severe, progressive neurodegeneratio...

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Bibliographic Details
Main Authors: Rachel Heon-Roberts, Annie L. A. Nguyen, Alexey V. Pshezhetsky
Format: Article
Language:English
Published: MDPI AG 2020-01-01
Series:Journal of Clinical Medicine
Subjects:
Online Access:https://www.mdpi.com/2077-0383/9/2/344