Lysosomal Storage Disease
Lysosomal storage diseases (LSDs) are a group of rare, inherited metabolic disorders characterized by the deficiency of specific lysosomal enzymes. This deficiency leads to the abnormal accumulation of undigested or partially digested macromolecules within lysosomes, causing cellular dysfunction and progressive damage to various organs and tissues. LSDs present with a wide spectrum of clinical manifestations affecting neurological, skeletal, visceral, and hematological systems, often leading to severe and life-limiting conditions.
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