Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration

• Главные авторы: Aryun Kim (Автор), Anita Grover (Автор), Kevin Hammon (Автор), Greg deHart (Автор), Peter Slasor (Автор), Anu Cherukuri (Автор), Temitayo Ajayi (Автор), David Jacoby (Автор), Angela Schulz (Автор), Nicola Specchio (Автор), Emily deLos Reyes (Автор), Paul Gissen (Автор), Joshua W. Henshaw (Автор)
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• Опубликовано: Wiley, 2021-03-01T00:00:00Z.
• Предметы: article
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Cerliponase alfa is recombinant human tripeptidyl peptidase 1 (TPP1) delivered by i.c.v. infusion for CLN2, a pediatric neurodegenerative disease caused by deficiency in lysosomal enzyme TPP1. We report the pharmacokinetics (PK) and pharmacodynamics of cerliponase alfa, the first i.c.v. enzyme repla...