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

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...

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Detalles Bibliográficos
Autores principales:	Kim, Aryun, Grover, Anita, Hammon, Kevin, de Hart, Greg, Slasor, Peter, Cherukuri, Anu, Ajayi, Temitayo, Jacoby, David, Schulz, Angela, Specchio, Nicola, de Los Reyes, Emily, Gissen, Paul, Henshaw, Joshua W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2020
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7993266/ https://www.ncbi.nlm.nih.gov/pubmed/33202105 http://dx.doi.org/10.1111/cts.12925

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