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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Библиографические подробности
Опубликовано в: :CPT Pharmacometrics Syst Pharmacol
Главные авторы: 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.
Формат: Artigo
Язык:Inglês
Опубликовано: John Wiley and Sons Inc. 2018
Предметы:
Research
Online-ссылка: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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