GENZ-112638 is primarily utilized in the field of medical research, particularly in the study and treatment of genetic disorders such as Gaucher disease, a lysosomal storage disorder caused by the accumulation of lipids in cells due to enzyme deficiency. It is often employed in preclinical studies to investigate its efficacy in modulating specific biochemical pathways implicated in these diseases. Researchers use this compound to explore its potential therapeutic benefits, especially through substrate reduction therapy to manage symptoms and improve patient outcomes. Additionally, GENZ-112638 is incorporated into experimental protocols to understand underlying cellular mechanisms and develop targeted therapies for Gaucher disease and other related conditions involving abnormal substance accumulation.