Tofersen treatment leads to sustained stabilization of disease in <scp>SOD1 ALS</scp> in a “real‐world” setting
This study provides evidence that tofersen treatment in SOD1 ALS can lead to meaningful preservation of function and suggestions of sustained improvement in neurologic function in some patients, and strongly supports the role of neurofilaments as therapeutic biomarkers.
Authors
- Sam Smith (ORCID: https://orcid.org/0000-0002-1488-7621)
- Jesse Markway
- Amber Malcolm
- Timothy M. Miller (ORCID: https://orcid.org/0000-0002-3424-5511)
- Robert C. Bucelli (ORCID: https://orcid.org/0000-0002-0063-8640)
- Kelly McCoy‐Gross
- Jeri Oranski
Institutions
- Washington University in St. Louis (US)
Publication Details
- Journal
- Annals of Clinical and Translational Neurology
- Published
- 2025-01-09
- DOI
- https://doi.org/10.1002/acn3.52264
- Citations
- 23
- Primary Topic
- Amyotrophic Lateral Sclerosis Research
- Type
- article
- Field-Weighted Citation Impact
- 31.95