Identifying biomarkers for Niemann-Pick Type C disease
Official title: Clinically Relevant Biomarkers for Niemann-Pick Type C
Funded at University of Illinois at Chicago · NIH-10931545 · Data updated 2026-11-12
This study is looking for helpful signs in the body that can show how Niemann-Pick Type C disease is changing over time and how well new treatments might work, which will ultimately help doctors better support patients and plan future studies.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Chicago NIH-funded |
| Lab location | 1 site (Chicago, UNITED STATES) |
| Project start | September 15, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $386,205 |
| NIH project number | 5U01FD008126-02 |
| Project ID | NIH-10931545 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Niemann-Pick Type C or those at risk due to family history.
✕Less likely to help
Patients with other lysosomal storage disorders or unrelated neurodegenerative conditions may not benefit from this research.
What this research studies
This research focuses on Niemann-Pick Type C (NPC), a genetic disorder that leads to progressive neurodegeneration and currently has no approved treatments. The team aims to identify and validate biomarkers that can provide insights into disease progression and therapeutic responses. By analyzing a larger cohort of biospecimens, they will prioritize biomarkers that reflect changes in the brain and other affected areas. This work is crucial for improving patient counseling and designing future clinical trials.
Why it matters
Potential benefit: If successful, this research could lead to better diagnostic tools and targeted therapies for patients with Niemann-Pick Type C.
How similar studies have gone: Previous research has shown promise in identifying biomarkers for other neurodegenerative diseases, suggesting potential success for this approach in NPC.
Where this research is happening
-
University of Illinois at Chicago
Chicago, United States Active
Researchers
- Principal investigator: Cologna, Stephanie M — University of Illinois at Chicago
- Study coordinator: Cologna, Stephanie 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.