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Keywords: substrate reduction
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Emerg Top Life Sci (2019) 3 (1): 63–73.
Published: 08 February 2019
..., is substrate reduction therapy (SRT). SRT addresses a common IEM phenotype associated with toxic accumulation of substrate from the defective enzyme, by inhibiting the formation of the substrate instead of directly repairing the defective enzyme. This minireview will summarize recent highlights...