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Cerliponase alfa (BMN 190) (formerly BMN-190)

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Company BioMarin Pharmaceutical Inc.
DescriptionRecombinant human tripeptidyl peptidase-1 (TPP1) enzyme replacement therapy
Molecular Target Tripeptidyl peptidase 1 (TPP1)
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Enzyme
Latest Stage of DevelopmentRegistration
Standard Indication Neurology (unspecified)
Indication DetailsTreat late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease
Regulatory Designation U.S. - Priority Review (Treat late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease)
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