Severe mucopolysaccharidosis type II (MPS II) is a progressive lysosomal storage disease
caused by mutations in the IDS gene, leading to a deficiency in the iduronate-2-sulfatase …

Reliable behavioural tests in animal models of neurodegenerative diseases allow us to
study the natural history of disease and evaluate the efficacy of novel therapies …

Mucopolysaccharidosis (MPS) type IIIA or Sanfilippo syndrome is a lysosomal storage
disorder characterised by progressive neurological pathology. Patients exhibit aggression …

Mucopolysaccharidosis (MPS) type I (Hurler syndrome) is a lysosomal storage disorder
characterized by deficiency of alpha-l-iduronidase (IDUA), intracellular storage of …

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

Mucopolysaccharidosis IIIB (MPS IIIB) is a lysosomal storage disorder characterized by
severe behavioural disturbances and progressive loss of cognitive and motor function …

The lysosomal storage pathology in Mucopolysaccharidosis (MPS) IIIB manifests in cells of
virtually all organs. However, it is the profound role of the neurological pathology that leads …

Abstract Background Mucopolysaccharidosis (MPS) IIIB, also known as Sanfilippo
Syndrome B, is a devastating childhood disease. Unfortunately, there are currently no …

Mucopolysaccharidosis type I (MPS-I or Hurler syndrome) is an inherited deficiency of the
lysosomal glycosaminoglycan (GAG)-degrading enzyme α-l-iduronidase (IDUA) in which …

Mucopolysaccharidosis type II (MPSII), or Hunter syndrome, is a devastating disorder
associated with a shortened life expectancy. Patients affected by MPSII have a variety of …