Tofersen, SOD1, and the Treatability of Amyotrophic Lateral Sclerosis

Affiliations

¹ Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia.
² Fixel Institute, Department of Neurology, University of Florida School of Medicine, Gainesville.
³ Department of Neurology, University of California, San Francisco.

Comment

Lauren Elman et al. JAMA Neurol. 2026.

. 2026 Feb 1;83(2):103-104.

doi: 10.1001/jamaneurol.2025.4927.

¹ Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia.
² Fixel Institute, Department of Neurology, University of Florida School of Medicine, Gainesville.
³ Department of Neurology, University of California, San Francisco.

No abstract available

Comment on

Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis.
Miller TM, Cudkowicz ME, Shaw PJ, Genge A, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Salachas F, Bruneteau G, Al-Chalabi A, Amorin M, Nestorov I, Graham D, Lin L, Sun P, McNeill M, Malek S, Inra J, Garafalo S, Fradette S; VALOR and OLE Working Group. Miller TM, et al. JAMA Neurol. 2026 Feb 1;83(2):115-125. doi: 10.1001/jamaneurol.2025.4946. JAMA Neurol. 2026. PMID: 41661214 Free PMC article. Clinical Trial.