Tofersen · Medicine question

How does tofersen reduce production of SOD1 protein?

iatroX medicinesPublished 8 November 2026

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Tofersen is an antisense oligonucleotide that binds to human SOD1 mRNA through complementary base pairing, triggering RNase-H-mediated degradation of the mRNA and thereby reducing SOD1 protein synthesis.

Sources for this answer

Qalsody 100mg solution for injectionBiogen Idec Ltd · emc · SmPC §5.1 Pharmacodynamic properties · Version 1Document updated 16 December 2025

Sources checked 8 November 2026

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