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Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis

29 Oct 2024

Abstract excerpt

Antisense oligonucleotides, which are used to silence target genes, are gaining attention as a novel drug discovery modality for proteinopathies. However, while clinical trials for neurodegenerative diseases like amyotrophic lateral sclerosis have been conducted in recent years, the results have not always been favorable. The results from a Phase III trial of the antisense oligonucleotide, that is, tofersen,...

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PMID: 39473221
DOI: 10.1002/acn3.52234

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