Ontology highlight
ABSTRACT:
SUBMITTER: Biliouris K
PROVIDER: S-EPMC6157691 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
CPT: pharmacometrics & systems pharmacology 20180816 9
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 cerebrospinal fluid (CSF), plasma, total spinal cord, brain, and pons. The estimated volumes were 13.6, 937, 4.5, 53.8, and 2.11 mL, respectively. Global sensitivity analysis demonstrated that the CSF-to- ...[more]