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Editorial
. 2025 Jun;71(6):928-931.
doi: 10.1002/mus.28413. Epub 2025 Apr 16.

Disease Modification in SOD1-ALS With Tofersen May Result in Serious CNS Inflammation

Stephen N Scelsa  1 , Daniel J L MacGowan  1
Affiliations
Editorial

Disease Modification in SOD1-ALS With Tofersen May Result in Serious CNS Inflammation

Stephen N Scelsa et al. Muscle Nerve. 2025 Jun.
No abstract available

Keywords: ALS; CNS inflammation; SOD1; Toferson; myelitis.

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References

    1. T. M. Miller, M. E. Cudkowicz, A. Genge, et al., “Trial of Antisense Oligonucleotide Tofersen for,” New England Journal of Medicine 387, no. 12 (2022): 1099–1110, https://doi.org/10.1056/NEJMoa2204705.
    1. T. Miller, M. Cudkowicz, P. J. Shaw, et al., “Phase 1‐2 Trial of Antisense Oligonucleotide Tofersen for,” New England Journal of Medicine 383, no. 2 (2020): 109–119, https://doi.org/10.1056/NEJMoa2003715.
    1. A. Lovett, S. Chary, S. Babu, et al., “Serious Neurologic Adverse Events in Tofersen Clinical Trials for Amyotrophic Lateral Sclerosis,” Muscle & Nerve 71, no. 6 (2025): 1006–1015. https://doi.org/10.1002/mus.28372.
    1. K. S. Frazier, “Antisense Oligonucleotide Therapies: The Promise and the Challenges From a Toxicologic Pathologist's Perspective,” Toxicologic Pathology 43, no. 1 (2015): 78–89, https://doi.org/10.1177/0192623314551840.
    1. L. Shen, J. A. Engelhardt, G. Hung, et al., “Effects of Repeated Complement Activation Associated With Chronic Treatment of Cynomolgus Monkeys With 2′‐O‐Methoxyethyl Modified Antisense Oligonucleotide,” Nucleic Acid Therapeutics 26, no. 4 (2016): 236–249, https://doi.org/10.1089/nat.2015.0584.

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