Biomarkers
Pilot synthesis. This page covers only the GBA-PD pilot range (
20240722_181748to20240722_181809, filed under gba-pd-asyn). The whole-section synthesis over the 27 sources in biomarkers-outcomes (Biomarker Validity / Validation / BMx Milestone Matrix / UPDRS / MDS-UPDRS / MoCA / NFL / RBD / Retina / SILK / Synapse / UPS / etc.) lives on biomarkers-outcomes. The two pages have disjoint source sets.
The GBA-PD pilot range treats biomarkers as a matrix across patient groups, therapeutic mechanism, target engagement, pathway modulation, and clinical readouts.
Seed Biomarker Set
| biomarker / readout | corpus context | source |
|---|---|---|
| GBA / GCase activity | GBA-PD, sPD, HC biomarker matrix; PR001 GCase activity and expression readouts. | 20240722_181748, 20240722_181805, 20240722_181809 |
| GlcCer | Biomarker matrix and therapeutic effect discussions. | 20240722_181748, 20240722_181756 |
| GlcSph | Biomarker matrix, venglustat/ambroxol discussion, and PR001 lipid accumulation evidence. | 20240722_181748, 20240722_181756, 20240722_181805 |
| Alpha-synuclein | Downstream pathology readout, including PR001 insoluble alpha-synuclein material. | 20240722_181805, 20240722_181809 |
| Cognitive readouts | Separate cognitive-readout table in the summary page. | 20240722_181748 |
Pilot Evidence Notes
| area | source-grounded detail |
|---|---|
| Sample-size logic | The pilot includes progression-to-MCI/dementia and GBA-activity-tertile scenarios with explicit sample-size estimates. Source: 20240722_181748. |
| GBA-PD / sPD / HC matrix | The source matrix separates GlcCer and GlcSph by brain vs CSF and by GBA-PD, sPD, and HC. It also flags data gaps for several gene-therapy/literature cells. Source: 20240722_181748. |
| LEAP lipid biomarkers | Venglustat LEAP notes include GlcCer and GlcSph LLOQ values in plasma/CSF and 52-week change-from-baseline values. Source: 20240722_181800. |
| MOVES-PD biomarkers | MOVES-PD notes include plasma/CSF GlcCer reduction, low or below-LLOQ GlcSph/lyso-GL-1 observations, NfL notes, DaTSCAN, and CSF total alpha-synuclein. Source: 20240722_181800. |
| Ambroxol biomarkers | Ambroxol clinical notes cover CSF/WBC GBA activity, CSF GBA protein, CSF alpha-synuclein, and cognitive/clinical endpoints in different trials. Sources: 20240722_181752, 20240722_181756. |
| PR001 biomarkers | PR001 pages include vector genomes, GCase activity/expression, GlcCer/GlcSph lipid accumulation, insoluble alpha-synuclein, astrogliosis/microgliosis, and NHP biodistribution. Sources: 20240722_181800, 20240722_181805, 20240722_181809. |
Open Structuring Questions
- Should lipid biomarkers get individual entity pages immediately, or wait until the full GBA-PD section is mapped?
- Should clinical endpoints live here or in a separate outcome-measures topic?
- Should uncertain source rows be promoted to a dedicated
questions/page?