Longitudinal Analysis of Superoxide Dismutase 1 Seeding Activity in Amyotrophic Lateral Sclerosis Cerebrospinal Fluid - PubMed
6 hours ago
- #Biomarkers
- #Amyotrophic Lateral Sclerosis
- #SOD1
- SOD1 seeding activity was detected in cerebrospinal fluid (CSF) of ALS patients, including those with and without SOD1 mutations, indicating misfolded SOD1 aggregates.
- In ALS progression, SOD1 seeding activity correlated with clinical decline measured by ALSFRS-R slope and increased CSF neurofilament light levels.
- SOD1 seeding activity was found early in the disease course in sporadic ALS patients, suggesting its potential as an early biomarker for clinical trial inclusion.
- The study highlights the potential of SOD1 seeding activity as a diagnostic, prognostic, and pharmacodynamic biomarker for SOD1-targeting therapies like tofersen, which could benefit a broader ALS population.