Long-Term Dynamics of CSF and Serum Neurofilament Light Chain in Adult Patients With 5q Spinal Muscular Atrophy Treated With Nusinersen
NfL decreased during nusinersen treatment, suggesting its potential as a pharmacodynamic response marker in adult SMA. However, in patients without detectable clinical improvement, our study cannot determine whether they represent a more sensitive outcome measure or are not clinically meaningful.
Authors
- René Günther (ORCID: https://orcid.org/0000-0003-0329-5644)
- Marcus Deschauer (ORCID: https://orcid.org/0000-0001-6116-6790)
- Cornelia Fuetterer (ORCID: https://orcid.org/0000-0002-8354-6660)
- Andreas Hahn (ORCID: https://orcid.org/0000-0003-0235-5211)
- Maren Freigang (ORCID: https://orcid.org/0000-0002-0941-6075)
- Annika Wachinger
- Tim Hagenacker (ORCID: https://orcid.org/0000-0002-3631-3450)
- Bogdan Bjelica (ORCID: https://orcid.org/0000-0002-1783-703X)
- Elke Hobbiebrunken (ORCID: https://orcid.org/0009-0003-3614-839X)
- Markus Weiler (ORCID: https://orcid.org/0000-0002-8942-7662)
- Anna Lisa Quinten
- Paul Lingor (ORCID: https://orcid.org/0000-0001-9362-7096)
- Svenja Brakemeier (ORCID: https://orcid.org/0000-0003-0678-0100)
- Jan Christoph Koch (ORCID: https://orcid.org/0000-0002-3778-0683)
- Isabell Cordts (ORCID: https://orcid.org/0000-0002-2078-1997)
- Emily Feneberg (ORCID: https://orcid.org/0000-0001-5905-3826)
- Bernhard Haller (ORCID: https://orcid.org/0000-0002-9723-393X)
- Tobias Keßler (ORCID: https://orcid.org/0000-0001-8350-7074)
- Susanne Petri
- Ricarda von Heynitz
Institutions
- German Cancer Research Center (DE)
- Heidelberg University (DE)
- Universitätsklinikum Gießen und Marburg (DE)
- University Hospital Heidelberg (DE)
- TUM Klinikum (DE)
- Medizinische Hochschule Hannover (DE)
- Universitätsmedizin Göttingen (DE)
- University Hospital Carl Gustav Carus (DE)
- Technical University of Munich (DE)
Publication Details
- Journal
- Neurology
- Published
- 2025-02-13
- DOI
- https://doi.org/10.1212/wnl.0000000000213371
- Citations
- 10
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 16.25
Funders
- Sanofi
- Biogen
- Teva Pharmaceutical Industries
- Cytokinetics
- Bundesministerium für Bildung und Forschung
- Ipsen
- Italfarmaco