Tofersen
Written and reviewed by Dr Ibrahim Imam, Consultant Neurologist. Last reviewed .
Tofersen. Tofersen is an antisense oligonucleotide (ASO). It is indicated for MND with SOD1 gene mutations. Fully referenced neurology checklist.
Pharmacology
- Tofersen is an antisense oligonucleotide (ASO)
- It is indicated for MND with SOD1 gene mutations
- It targets the SOD1 mRNA
- It may trigger an immune inflammatory response
Dosing
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Adverse events
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Outcomes
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References
- Miller T, Cudkowicz M, Shaw PJ, et al. Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med 2020; 383:109-119.
- Miller TM, Cudkowicz ME, Genge A, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med 2022; 387:1099-1110.
- Blair HA. Tofersen: first approval. Drugs 2023 (Online ahead of print).
- Simonini C, Zucchi E, Martinelli I, et al. Neurodegenerative and neuroinflammatory changes in SOD1-ALS patients receiving tofersen. Sci Rep 2025; 15:11034.
- McGuigan A, Blair HA. Tofersen: a review in amyotrophic lateral sclerosis associated with SOD1 mutations. CNS Drugs 2025; 39:903-912.
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