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Tag: Acid Sphingomyelinase Deficiency


FDA, Regulations

FDA approves first treatment for Acid Sphingomyelinase Deficiency, a rare genetic disease

August 31, 2022

Via: World Pharma News

Today, the U.S. Food and Drug Administration approved Xenpozyme (Olipudase alfa) for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD), a rare genetic disease that causes premature death. Xenpozyme is the first approved medication to treat […]