CSF and plasma GFAP and VEGF in adult type 3 spinal muscular atrophy patients treated with nusinersen

Key Points

Sustained motor function improvements were observed in adult type 3 spinal muscular atrophy patients treated with nusinersen, despite nonsignificant biomarker trajectories.
Over a 30-month period, both cerebrospinal fluid and plasma levels of GFAP and VEGF were monitored during nusinersen treatment.
Analysis of biomarker trajectories was performed, aiming to enhance understanding of SMA pathogenesis and treatment effects.
Future research in larger, collaborative cohorts may uncover further insights into the role of biomarkers in spinal muscular atrophy.

Abstract

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.

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Cite This Study

Pronto‐Laborinho et al. (Thu,) studied this question.

synapsesocial.com/papers/69a767c8badf0bb9e87e250e https://doi.org/https://doi.org/10.1016/j.jns.2026.125795

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