What is venglustat? An Oral Investigational Substrate Reduction Therapy
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4 min read
Initially developed by Genzyme and later acquired by Sanofi, venglustat is an investigational oral medication functioning as a substrate reduction therapy. This small-molecule compound has been explored for treating several rare lysosomal storage disorders, including Fabry and Gaucher disease, by targeting the synthesis of harmful glycosphingolipids.
