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Tofersen and other antisense oligonucleotides in ALS

1 Jan 2025

Abstract excerpt

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 to be the paradigm disease in dominant degenerative diseases. The results of treatment with the ASO tofersen in...

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PMID: 39845577
DOI: 10.1177/17562864251313915

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