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

A Review on Spinal Muscular Atrophy: Awareness, Knowledge, and Attitudes

(2016) Rebecca R. Moultrie et al.   Journal of Genetic Counseling

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

(2016) Claudia A. Chiriboga et al.   NEUROLOGY

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy

(2015) Laurent P. Bogdanik et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Pharmacokinetic–pharmacodynamic modeling for reduction of hepatic apolipoprotein B mRNA and plasma total cholesterol after administration of antisense oligonucleotide in mice

(2014) Ryosuke Shimizu et al.   JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS

Prediction-Corrected Visual Predictive Checks for Diagnosing Nonlinear Mixed-Effects Models

(2011) Martin Bergstrand et al.   AAPS Journal

Spinal muscular atrophy

(2011) Adele D'Amico et al.   Orphanet Journal of Rare Diseases

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT

Understanding pharmacokinetics using realistic computational models of fluid dynamics: biosimulation of drug distribution within the CSF space for intrathecal drugs

(2010) Andreas Kuttler et al.   JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS

Increased fat mass and high incidence of overweight despite low body mass index in patients with spinal muscular atrophy

(2009) Douglas M. Sproule et al.   NEUROMUSCULAR DISORDERS