Background: Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative
disorder characterized by degeneration of spinal motor neurons leading to muscular …

Background: Nusinersen is an antisense-oligonucleotide (ASO) approved for treatment of 5q-
spinal muscular atrophy (SMA). Since the drug cannot cross the blood-brain barrier (BBB), it …

Spinal muscular atrophy (SMA) is a progressive motor neuron disease with onset during
infancy or early childhood. Recent therapeutic advances targeting the genetic defect that …

Approval of nusinersen, an intrathecally administered antisense oligonucleotide, for the
treatment of 5q-spinal muscular atrophy (SMA) marked the beginning of a new therapeutic …

Abstract Introduction/Aims There is limited information on the potential effects of repeated
intrathecal antisense oligonucleotide drug delivery on cerebrospinal fluid (CSF) biochemical …

Background: The antisense oligonucleotide Nusinersen recently became the first approved
drug against spinal muscular atrophy (SMA). It was approved for all ages, albeit the clinical …

The antisense oligonucleotide Nusinersen has been recently licensed to treat spinal
muscular atrophy (SMA). Since SMA type 3 is characterized by variable phenotype and …

Background Nusinersen is an intrathecally administered antisense oligonucleotide (ASO)
that improves motor function in patients with spinal muscular atrophy (SMA). In addition to …

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

Spinal muscular atrophy is a genetic motor neuron disease that leads to progressive
muscular atrophy and muscle weakness. In December 2016, the Food and Drug …