Biomarker Discovery for Batten Disease
Official title: Research Project 2
Funded at Hugo W. Moser Res Inst Kennedy Krieger · NIH-11417535 · Data updated 2026-11-12
Researchers are developing biomarkers to track disease progression and treatment response in children with Batten disease.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Hugo W. Moser Res Inst Kennedy Krieger NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | September 15, 2025 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $143,917 |
| NIH project number | 5U54HD122210-02 |
| Project ID | NIH-11417535 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children affected by Batten disease or related NCLs, with potential inclusion for biomarker sampling and longitudinal tracking.
✕Less likely to help
Some patients may not experience direct benefit if biomarker development does not translate into available therapies during the study period.
What this research studies
This project aims to uncover reliable molecular, imaging, and physiological biomarkers that can be measured in patients with Batten disease to monitor how the disease changes over time and in response to therapies. Using high-content omics approaches, the team plans to build a panel of disease-specific biomarkers and a biosignature score that reflects Batten disease biology. The work focuses on making these biomarkers minimally invasive and suitable for use in future clinical trials to evaluate new treatments. By aligning biomarker endpoints with disease pathways, the study seeks to improve trial readiness and regulatory support for patient-focused therapies.
Why it matters
Potential benefit: If successful, patients could benefit from more precise monitoring of disease progression and faster assessment of treatment effects in future trials.
How similar studies have gone: Prior efforts in NCLs have shown biomarker potential, with ongoing work suggesting feasibility of biomarker-based monitoring, though this project advances toward a comprehensive biomarker panel.
Where this research is happening
-
Hugo W. Moser Res Inst Kennedy Krieger
Baltimore, United States Active
Researchers
- Principal investigator: Weimer, Jill M — Hugo W. Moser Res Inst Kennedy Krieger
- Study coordinator: Weimer, Jill M
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.