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

• Main Authors: Konstantinos Biliouris (Author), Puneet Gaitonde (Author), Wei Yin (Author), Daniel A. Norris (Author), Yanfeng Wang (Author), Scott Henry (Author), Robert Fey (Author), Ivan Nestorov (Author), Stephan Schmidt (Author), Mark Rogge (Author), Lawrence J. Lesko (Author), Mirjam N. Trame (Author)
• Format: Book
• Published: Wiley, 2018-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...