Wednesday, August 31, 2022

US FDA News: FDA Approves First Treatment for Acid Sphingomyelinase Deficiency, a Rare Genetic Disease

The FDA 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.
Read more: FDA Approves First Treatment for Acid Sphingomyelinase Deficiency, a Rare Genetic Disease