The landscape of treating Mucopolysaccharidosis Type I has experienced remarkable advancement, evolving from an era of limited therapeutic options to a sophisticated array of treatment modalities that target the disease at multiple levels. This rare lysosomal storage disorder, characterized by alpha-L-iduronidase deficiency, presents unique clinical challenges that have catalyzed...

  Understanding the Molecular Foundation of MPS The complexity of Mucopolysaccharidosis (MPS) stems from fundamental disruptions in cellular metabolism that affect glycosaminoglycan processing. These inherited disorders create a domino effect within cells, where defective lysosomal enzymes fail to properly degrade complex carbohydrate structures, resulting in toxic accumulation that...