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Synthetic enzyme inhibitors are not only effective in elucidating the physiological functions of complex carbohydrates but also effective in treating diseases that occur due to the excessive accumulation of specific glycan chains. Gaucher’s disease, a type of lysosomal disease, presents with hepatosplenomegaly and central nervous system symptoms due to the accumulation of glucosylceramide (GlcCer) caused by a genetic deficiency of glucocerebrosidase. Eliglustat (Fig. 117.1), a GlcCer synthase inhibitor, has been confirmed to be effective as a substrate reduction therapy (SRT) and is used as a drug for the treatment of patients with type 1 Gaucher’s disease [1, 2].

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Synthetic Enzyme Inhibitors (Eliglustat)

  • 章节

  • 发表日期：

    2026年

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  • Jin-ichi Inokuchi

摘要

Synthetic enzyme inhibitors are not only effective in elucidating the physiological functions of complex carbohydrates but also effective in treating diseases that occur due to the excessive accumulation of specific glycan chains. Gaucher’s disease, a type of lysosomal disease, presents with hepatosplenomegaly and central nervous system symptoms due to the accumulation of glucosylceramide (GlcCer) caused by a genetic deficiency of glucocerebrosidase. Eliglustat (Fig. 117.1), a GlcCer synthase inhibitor, has been confirmed to be effective as a substrate reduction therapy (SRT) and is used as a drug for the treatment of patients with type 1 Gaucher’s disease [1, 2].