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LSD treatments may involve hematopoietic stem cell transplantation (HSCT) to replenish active enzymes, small molecule-based substrate reduction therapies (SRTs) to decrease the production of accumulating substrates, pharmacological chaperone therapies that stabilize mutated enzymes, enzyme replacement therapies (ERTs), and gene therapy
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Color:Chartreuse Pikey 528-30125
9 of Mallon et al
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