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

Abstract excerpt

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 neurodegeneration but mild somatic involvement, with patients losing milestones and previously acquired skills as the disease...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Cellular transport and secretion
• Share to Lysosomal Storage Disorders Research
• Share to Trypanosoma species research and implications