Tofersen and other antisense oligonucleotides in ALS

The advent of antisense oligonucleotide (ASO) therapies in neurodegenerative disorders is associated with enormous hope. Nusinersen treatment was a breakthrough intervention in the recessive disease spinal muscular atrophy, and superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) seems...

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Bibliographic Details
Main Authors: Albert Ludolph, Maximilian Wiesenfarth
Format: Article
Language:English
Published: SAGE Publishing 2025-01-01
Series:Therapeutic Advances in Neurological Disorders
Online Access:https://doi.org/10.1177/17562864251313915
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