Targeted Proteomics upon Treatment with Tofersen Identifies Novel Response Markers for Superoxide Dismutase 1‐Linked Amyotrophic Lateral Sclerosis
Our results highlight a complex, time-dependent differential response of CSF biomarkers to tofersen treatment, and may pave the way for developing a panel of responsive proteins to make biomarker endpoints more robust in clinical trials for SOD1-ALS and beyond. ANN NEUROL 2025;98:1318-1334.
Authors
- René Günther (ORCID: https://orcid.org/0000-0003-0329-5644)
- Maximilian Vidovic (ORCID: https://orcid.org/0000-0003-1453-1681)
- Dávid Brenner (ORCID: https://orcid.org/0000-0002-1535-3146)
- Thomas Haarmeier
- Peter Reilich (ORCID: https://orcid.org/0000-0002-2464-0681)
- Patrick Weydt (ORCID: https://orcid.org/0000-0003-2344-5662)
- Alberto Catanese (ORCID: https://orcid.org/0000-0002-8266-4913)
- Tim Hagenacker (ORCID: https://orcid.org/0000-0002-3631-3450)
- Simon Witzel (ORCID: https://orcid.org/0000-0003-1841-954X)
- Patrick Oeckl (ORCID: https://orcid.org/0000-0002-7652-7023)
- Paul Lingor (ORCID: https://orcid.org/0000-0001-9362-7096)
- Željko Uzelac (ORCID: https://orcid.org/0000-0003-2479-2025)
- Maximilian Wiesenfarth (ORCID: https://orcid.org/0009-0004-0187-5973)
- Joachim Schuster (ORCID: https://orcid.org/0000-0002-4207-1312)
- Andreas Hermann (ORCID: https://orcid.org/0000-0002-7364-7791)
- Jochen Hans Weishaupt (ORCID: https://orcid.org/0000-0002-3399-9784)
- Hayrettin Tumani (ORCID: https://orcid.org/0000-0002-7921-9966)
- Zeynep Elmas
- Ritchie Ho (ORCID: https://orcid.org/0000-0003-1496-4436)
- Johannes Prudlo (ORCID: https://orcid.org/0000-0002-7932-5929)
- Kornelia Günther
- Alexander German (ORCID: https://orcid.org/0000-0002-8708-6809)
- Wolfgang Ruf (ORCID: https://orcid.org/0000-0002-8105-6123)
- Ute Weyen
- Julian Großkreutz (ORCID: https://orcid.org/0000-0001-9525-1424)
- Antje Knehr
- Martin Regensburger (ORCID: https://orcid.org/0000-0002-2172-7386)
- Axel Freischmidt (ORCID: https://orcid.org/0000-0003-1216-3790)
- Franziska Bachhuber (ORCID: https://orcid.org/0000-0003-1031-9966)
- Thomas Meyer (ORCID: https://orcid.org/0000-0003-3194-7347)
- Christina Steffke
- Karthik Baskar
- Susanne Petri
- Albert C. Ludolph
- Johannes Dorst
- Florian Schöberl
- Joachim Wolf
Institutions
- Cedars-Sinai Medical Center (US)
- University of Bonn (DE)
- University of Freiburg (DE)
- University of Mannheim (DE)
- Friedrich-Alexander-Universität Erlangen-Nürnberg (DE)
- Universität Ulm (DE)
- Heidelberg University (DE)
- German Center for Neurodegenerative Diseases (DE)
- University Hospital Heidelberg (DE)
- TUM Klinikum (DE)
- Medizinische Hochschule Hannover (DE)
- Humboldt-Universität zu Berlin (DE)
- Institute of Pathology Celle (DE)
- Helios Klinikum Krefeld (DE)
- Munich Cluster for Systems Neurology (DE)
- Essen University Hospital (DE)
- Diakonissenkrankenhaus (DE)
- Friedrich Baur Stiftung (DE)
- BG University Hospital Bergmannsheil Bochum (DE)
- University Hospital Ulm (DE)
- University Hospital Carl Gustav Carus (DE)
- Technische Hochschule Ulm (DE)
- University of Rostock (DE)
- Ludwig-Maximilians-Universität München (DE)
- Ruhr University Bochum (DE)
- University of Lübeck (DE)
Publication Details
- Journal
- Annals of Neurology
- Published
- 2025-08-09
- DOI
- https://doi.org/10.1002/ana.70025
- Citations
- 5
- Primary Topic
- Amyotrophic Lateral Sclerosis Research
- Type
- article
- Field-Weighted Citation Impact
- 6.95
Funders
- Deutsche Forschungsgemeinschaft
- Else Kröner-Fresenius-Stiftung
- Deutsche Gesellschaft für Muskelkranke