Antisense oligonucleotides (ASOs) are short oligodeoxynucleotides designed to bind to
specific regions of target mRNA. ASOs can modulate pre-mRNA splicing, increase levels of …

Antisense oligonucleotides (ASOs) are short, synthetic, single‐stranded deoxynucleotide
sequences composed of phosphate backbone‐connected sugar rings. Designing of those …

Abstract Introduction/Aims In amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations
(SOD1‐ALS), tofersen received accelerated approval in the United States and is available …

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease affecting the brain
and spinal cord motor neurons. On 25 April 2023, the drug tofersen, an antisense …

Amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, is a motor neuron disease. In
ALS, upper and lower motor neurons in the brain and spinal cord progressively degenerate …

Introduction Amyotrophic lateral sclerosis (ALS) is a rapidly progressive motor neuron
disorder with a fatal outcome 3–5 years after disease onset due to respiratory complications …

Abstract Background In Amyotrophic Lateral Sclerosis (ALS) patients with SOD1 mutation
the intrathecal administration of tofersen slowed down the progression of disease in a …

This review offers an in-depth examination of amyotrophic lateral sclerosis (ALS),
addressing its epidemiology, pathophysiology, clinical presentation, diagnostic techniques …

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease mainly
characterized by the accumulation of ubiquitinated proteins in the affected motor neurons. At …

Objective Patients with amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase
1 (SOD1) gene mutations (SOD1 ALS) treated with tofersen have shown slowing of disease …