Abstract
Mucopolysaccharidosis type I (MPS I) is a genetic disorder in which there is a deficiency in alpha-L-iduronidase (IDUA gene) enzyme expression, responsible for the lysosomal degradation of two glycosaminoglycans. Lysosomal disturbance caused by glycosaminoglycans accumulation triggers secondary effects among which gangliosides GM2, GM3 and GD3 storage on central nervous system (CNS), invo…