The Mutation Matters: <scp>CSF</scp> Profiles of <scp>GCase</scp>, Sphingolipids, α‐Synuclein in <scp>PD_GBA</scp>

Abstract

These findings confirm that GBA mutations have a relevant functional impact on biomarker profiles in patients. Bridging the gap between genetics and biochemical profiles now allows patient stratification for clinical trials merely based on mutation status. Importantly, all findings were most prominent in PD_GBA with severe variants. © 2021 International Parkinson and Movement Disorder Society.