A Semi‐Mechanistic Population Pharmacokinetic Model of Nusinersen: An Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy

A pharmacokinetic (PK) model was developed for nusinersen, an antisense oligonucleotide (ASO) that is the first approved treatment for spinal muscular atrophy (SMA). The model was built with data from 92 nonhuman primates (NHPs) following intrathecal doses (0.3–7 mg) and characterized the PK in cere...

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Detalles Bibliográficos
Publicado en:CPT Pharmacometrics Syst Pharmacol
Main Authors: Biliouris, Konstantinos, Gaitonde, Puneet, Yin, Wei, Norris, Daniel A., Wang, Yanfeng, Henry, Scott, Fey, Robert, Nestorov, Ivan, Schmidt, Stephan, Rogge, Mark, Lesko, Lawrence J., Trame, Mirjam N.
Formato: Artigo
Idioma:Inglês
Publicado: John Wiley and Sons Inc. 2018
Assuntos:
Research
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6157691/
https://ncbi.nlm.nih.gov/pubmed/30043511
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/psp4.12323
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