Serious Neurologic Adverse Events in Tofersen Clinical Trials for Amyotrophic Lateral Sclerosis
Some antisense oligonucleotides (ASOs) have been described as having pro-inflammatory properties. Aseptic meningitis has been reported with nusinersen; however, myelitis, radiculitis, increased intracranial pressure, and papilledema have not been reported with ASO treatment. These neurologic AEs should be considered when assessing the overall benefit/risk of tofersen treatment for SOD1-ALS. Safety data from the open-label extension and expanded access program will continue to characterize these events and further inform the safety profile of tofersen in SOD1-ALS.
Authors
- Sohail Malek
- Gaëlle Bruneteau (ORCID: https://orcid.org/0000-0002-1905-7985)
- Keith Mayl (ORCID: https://orcid.org/0009-0002-6581-7567)
- Toby A. Ferguson (ORCID: https://orcid.org/0000-0003-3938-2280)
- Thos Cochrane
- Laura Fanning (ORCID: https://orcid.org/0000-0002-2685-3875)
- Karen Smirnakis
- Jennifer Inra
- Suma Babu (ORCID: https://orcid.org/0000-0002-2648-7625)
- Stephanie Fradette
- Sowmya Chary
- Christopher McDermott (ORCID: https://orcid.org/0000-0002-3857-446X)
- Shafeeq Ladha (ORCID: https://orcid.org/0000-0003-1955-4292)
- Merete Karlsborg
- Robert C. Bucelli (ORCID: https://orcid.org/0000-0002-0063-8640)
- Adriano Chiò
- Jonathan D. Glass
- Alexandra Lovett
Institutions
- Biogen (United States) (US)
- Barrow Neurological Institute (US)
- Harvard University (US)
- Emory University (US)
- King's College London (GB)
- Washington University in St. Louis (US)
- Bispebjerg Hospital (DK)
- Sorbonne Université (FR)
- Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT)
- Massachusetts General Hospital (US)
- Assistance Publique – Hôpitaux de Paris (FR)
- Sheffield Teaching Hospitals NHS Foundation Trust (GB)
- King's College Hospital NHS Foundation Trust (GB)
- Pitié-Salpêtrière Hospital (FR)
- Institut du Cerveau (FR)
- University of Turin (IT)
- University of Sheffield (GB)
Publication Details
- Journal
- Muscle & Nerve
- Published
- 2025-02-27
- DOI
- https://doi.org/10.1002/mus.28372
- Citations
- 22
- Primary Topic
- Amyotrophic Lateral Sclerosis Research
- Type
- article
- Field-Weighted Citation Impact
- 29.17
Funders
- Biogen