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olipudase alfa (GZ402665)

Also known as: formerly rhASM

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Company Sanofi
DescriptionRecombinant human acid sphingomyelinase (rhASM)
Molecular Target
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Enzyme
Latest Stage of DevelopmentPhase I/II
Standard IndicationGlycosphingolipid storage disorders
Indication DetailsTreat Niemann-Pick type B disease
Regulatory Designation U.S. - Breakthrough Therapy (Treat Niemann-Pick type B disease)
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