CSF and plasma GFAP and VEGF in adult type 3 spinal muscular atrophy patients treated with nusinersen
CSF and plasma GFAP and VEGF trajectories over a 30-month period under nusinersen treatment were nonsignificant. This study confirms sustained motor function improvements in adult type 3 SMA patients treated with nusinersen. Future research in larger, multicentric, and internationally collaborative cohorts are essential to fully understand the role of these potential biomarkers in SMA pathogenesis.
Authors
- Ana Catarina Pronto‐Laborinho (ORCID: https://orcid.org/0000-0002-2816-9698)
- M. Viseu De Carvalho
- Miguel Oliveira Santos (ORCID: https://orcid.org/0009-0007-0753-9200)
- Sara Domingues (ORCID: https://orcid.org/0000-0002-8649-9038)
- Teresa Freitas
- Michele Gomes Rosa
Institutions
- University of Lisbon (PT)
- Iscte – Instituto Universitário de Lisboa (PT)
- Hospital de Santa Maria (PT)
Publication Details
- Journal
- Journal of the Neurological Sciences
- Published
- 2026-02-05
- DOI
- https://doi.org/10.1016/j.jns.2026.125795
- Citations
- 2
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 73.86
Funders
- Biogen