Tailoring Treatment to the Type of Mucopolysaccharidosis
Mucopolysaccharidosis (MPS) constitutes a collection of rare inherited metabolic conditions stemming from specific lysosomal enzyme deficiencies that compromise glycosaminoglycan (GAG) catabolism. Impaired enzymatic function leads to progressive GAG accumulation, resulting in multisystem pathology affecting organs, skeletal structures, and connective tissues. Deepening comprehension of...
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