Olipudase Alfa
CAS 927883-84-9Biologic
Overview
Olipudase Alfa is a recombinant human enzyme replacement therapy for the treatment of acid sphingomyelinase deficiency, which leads to Niemann-Pick disease types A and B. It works by replacing the deficient enzyme, thereby helping to reduce sphingomyelin accumulation in organs, particularly the liver and spleen.
Therapeutic areas
Lysosomal Storage DisordersNiemann-Pick Disease
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