[HTML][HTML] Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses

EG Shapiro, ML Escolar, KA Delaney… - Molecular Genetics and …, 2017 - Elsevier
The mucopolysaccharidoses (MPS) are a group of rare, inherited lysosomal storage
disorders in which accumulation of glycosaminoglycans (GAGs) leads to progressive tissue
and organ dysfunction. In addition to a variety of somatic signs and symptoms, patients with
rapidly progressing MPS I (Hurler), II, III, and VII can present with significant neurological
manifestations, including impaired cognitive abilities, difficulties in language and speech,
behavioral abnormalities, sleep problems, and/or seizures. Neurological symptoms have a …