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Olipudase Alfa


Drug Names(s): GZ402665, Recombinant Acid Sphingomyelinase, rhASM, SAR402665

Description: Olipudase alfa is a recombinant human acid sphingomyelinase. Acid sphingomyelinases catalyze the breakdown of sphingomyelin to ceramide and phosphorylcholine. Lack of the enzyme causes Niemann–Pick disease (NPD) types A (a neuronopathic form) and B (a nonneuronopathic form).

Since olipudase does not cross the blood brain barrier, it is being developed for Niemann-Pick type B disease, where non-neurologic symptoms predominate.

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