The mucopolysaccharidoses (MPSs) are inherited metabolic disorders (IMDs) caused by
single-gene defects leading to progressive cellular accumulation of glycosaminoglycans
(GAGs) and damage to multiple organs, including the central nervous, musculoskeletal,
cardiorespiratory, and other systems. Hurler syndrome (MPS IH), the most severe form, is the
prototypical model. Enzyme replacement therapy (ERT), available for MPS I, II, and VI, is
beneficial in some patients. However, ERT does not improve neurocognitive function …