FDA Approves First Treatment for Acid Sphingomyelinase Deficiency, a Rare Genetic Disease
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 symptoms that are not related to the central nervous system in patients with ASMD. “ASMD … Continue reading FDA Approves First Treatment for Acid Sphingomyelinase Deficiency, a Rare Genetic Disease
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