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...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Udgivet i:Clin Transl Sci
Main Authors: 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.
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2020
Fag:
Research
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7993266/
https://ncbi.nlm.nih.gov/pubmed/33202105
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/cts.12925
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker