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BioWorld - Friday, February 13, 2026
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Home » Two enzymatic modulators could alleviate Gaucher disease
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Endocrine/metabolic

Two enzymatic modulators could alleviate Gaucher disease

Aug. 26, 2024
By Mar de Miguel
People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases.
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