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