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Article

Tofersen in SOD1-associated amyotrophic lateral sclerosis: From molecular mechanisms to regulatory milestones.

European journal of pharmaceutical sciences : official journal of the European Federation for Pharmaceutical Sciences - 1 Aug 2026

Braza Antonio J, Viñas-Bastart Montserrat, Sureda-Rosich Maria, García-Parra Beliu, Guiu-Segura Josep M, Modamio Pilar

Abstract excerpt

BACKGROUND: Amyotrophic Lateral Sclerosis (ALS) is a progressive and ultimately fatal neurodegenerative disorder characterized by degeneration of upper and lower motor neurons. Mutations in the superoxide dismutase 1 (SOD1) gene account for approximately 2% of ALS cases and are associated with toxic protein misfolding and aggregation. Tofersen is an antisense oligonucleotide therapy designed to reduce the...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 42173382
DOI: 10.1016/j.ejps.2026.107563

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SOD1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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