The neurons of the central nervous system (CNS) require precise control of their bathing
microenvironment for optimal function, and an important element in this control is the blood …

Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic
diseases caused by functional defects in specific lysosomal proteins. The lysosome is a …

Mucopolysaccharidoses (MPSs) constitute a heterogeneous group of lysosomal storage
disorders characterized by the lysosomal accumulation of glycosaminoglycans (GAGs) …

Lysosomal storage diseases (LSDs) are a group of rare, life-threatening genetic disorders,
usually caused by a dysfunction in one of the many enzymes responsible for intralysosomal …

Mucopolysaccharide diseases (MPS) are caused by deficiency of glycosaminoglycan (GAG)
degrading enzymes, leading to GAG accumulation. Neurodegenerative MPS diseases …

Neurodegenerative diseases are caused by the progressive loss of function or structure of
nerve cells in the central nervous system. The most common neurodegenerative diseases …

The majority of lysosomal storage diseases affect the brain. Treatment of the brain with
intravenous enzyme replacement therapy is not successful, because the recombinant …

Mucopolysaccharidoses (MPS) are inborn errors of metabolism produced by a deficiency of
one of the enzymes involved in the degradation of glycosaminoglycans (GAGs). Although …

Neuroinflammation is indicated in the pathogenesis of several acute and chronic
neurological disorders. Acute lesions in the brain parenchyma induce intense and highly …

Mucopolysaccharidosis III is a rare genetic disease characterized by progressive cognitive
decline and severe hyperactivity that does not respond to stimulants. Somatic features are …