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Article

Tofersen: A Review in Amyotrophic Lateral Sclerosis Associated with SOD1 Mutations.

CNS drugs - 1 Sept 2025

McGuigan Aisling, Blair Hannah A

Abstract excerpt

Tofersen (QALSODY®) is the first drug approved for the treatment of amyotrophic lateral sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) mutations. Tofersen is an antisense oligonucleotide that induces SOD1 mRNA degradation. In the 28-week, placebo-controlled, multinational, phase III VALOR trial, intrathecally administered tofersen reduced plasma concentrations of neurofilament proteins (biomarker...

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Share to Amyotrophic Lateral Sclerosis
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 40640528
DOI: 10.1007/s40263-025-01204-5

Genes

SOD1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026.

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