Spinal muscular atrophy (SMA) is a progressive degenerative illness that affects 1 in every 6
to 11,000 live births. This autosomal recessive disorder is caused by homozygous deletion …

Summary Background In April 2023, the antisense oligonucleotide tofersen was approved
by the US Food and Drug Administration (FDA) for treatment of SOD1-amyotrophic lateral …

Cerebrospinal fluid (CSF) diagnostics has emerged as a valid tool for a variety of
neurological diseases. However, CSF diagnostics has been playing a subordinate role in …

Objective To investigate biomarkers of disease progression in cerebrospinal fluid (CSF) and
serum in adult patients with spinal muscular atrophy (SMA). Furthermore, we assess the …

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

Fragile X-associated Tremor/Ataxia Syndrome (FXTAS) is a neurodegenerative disorder
associated with the FMR1 premutation. Currently, it is not possible to determine when and if …

Background Studies regarding the impact of (neuro) inflammation and inflammatory
response following repetitive, intrathecally administered antisense oligonucleotides (ASO) in …

Spinal muscular atrophy is a devastating motor neuron disease characterized by severe
cases of fatal muscle weakness. It is one of the most common genetic causes of mortality …

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 …

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