Olipudase Alfa

CAS 927883-84-9 1 FDA-DMF supplierBiologic

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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