Population Pharmacokinetics of Nusinersen in the Cerebral Spinal Fluid and Plasma of Pediatric Patients With Spinal Muscular Atrophy Following Intrathecal Administrations

Population Pharmacokinetics of Nusinersen in the Cerebral Spinal Fluid and Plasma of Pediatric Patients With Spinal Muscular Atrophy Following Intrathecal Administrations …………… Luu KT, Norris DA, Gunawan R, Henry S, Geary R, Wang Y. J Clin Pharmacol. 2017 Aug;57(8):1031-1041. PubMed 00

Lack of QT Prolongation for 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides Based on Retrospective Exposure/Response Analysis of Ten Phase 1 Dose-Escalation Placebo-Controlled Studies in Healthy Subjects

Lack of QT Prolongation for 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides Based on Retrospective Exposure/Response Analysis of Ten Phase 1 Dose-Escalation Placebo-Controlled Studies in Healthy Subjects …………… Yu RZ, Gunawan R, Geary RS, Hughes SG, Henry SP, Wang Y. Nucleic Acid Ther. 2017 Aug 11. PubMed 00