Objective: To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy
of intrathecal nusinersen (previously ISIS-SMNRx), an antisense oligonucleotide designed …

Background Nusinersen is an antisense oligonucleotide drug that modulates pre–
messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been …

Objective To report results of intrathecal nusinersen in children with later-onset spinal
muscular atrophy (SMA). Methods Analyses included children from a phase 1b/2a study …

Background Nusinersen is a 2′-O-methoxyethyl phosphorothioate-modified antisense drug
being developed to treat spinal muscular atrophy. Nusinersen is specifically designed to …

Objective To identify the level of evidence for use of nusinersen to treat spinal muscular
atrophy (SMA) and review clinical considerations regarding use. Methods The author panel …

Background Treatment with nusinersen has demonstrated significant and clinically
meaningful benefits in clinical trials in infants and children with spinal muscular atrophy …

Abstract Introduction The EMBRACE study (Clinical Trials No. NCT02462759) evaluated
nusinersen in infants/children with infantile‐or later‐onset spinal muscular atrophy (SMA) …

Spinal muscular atrophy (SMA) encompasses a group of autosomal recessively inherited
degenerative neuromuscular disorders. They range in severity from neonatal onset with …

Spinal muscular atrophy (SMA) is a rare autosomal recessive disorder characterized by
muscle atrophy and weakness resulting from motor neuron degeneration in the spinal cord …

Background: Nusinersen is an intrathecally administered antisense oligonucleotide (ASO)
and the first approved drug for the treatment of spinal muscular atrophy (SMA). However …