Biomarkers and clinical tools for Sanfilippo syndrome type C (MPS IIIC)
Official title: Identification of Potential Biomarkers and Clinical Tools for use in Mucopolysaccharidosis IIIC Patients.
Funded at Phoenix Nest, INC. · NIH-11128545 · Data updated 2026-11-12
Create new lab tests and clinical tools to help detect and follow MPS IIIC (Sanfilippo type C) in affected children and adults.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Phoenix Nest, INC. NIH-funded |
| Lab location | 1 site (Brooklyn, United States) |
| Project start | July 1, 2024 |
| Project end | June 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $1,062,505 |
| NIH project number | 5R44NS137892-02 |
| Project ID | NIH-11128545 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children, teens, or adults with confirmed or suspected MPS IIIC and caregivers willing to provide biological samples and medical history are the ideal candidates.
✕Less likely to help
People without MPS IIIC (for example those with other types of MPS or unrelated conditions) are unlikely to receive direct benefit from these specific biomarkers.
What this research studies
If you join this project, the team will work to find measurable signs in blood, urine, or other samples that link to MPS IIIC. They will collect samples and clinical information from people with confirmed or suspected MPS IIIC and compare them to controls. The researchers will develop laboratory assays and clinical tools and test whether these markers reliably track disease status or progression. Successful markers could be used to help doctors monitor symptoms and to support future treatment trials.
Why it matters
Potential benefit: These tests could allow earlier diagnosis and better tracking of disease progression, helping guide care and future treatments for people with MPS IIIC.
How similar studies have gone: Some biomarker work has been done for other Sanfilippo subtypes, but validated MPS IIIC-specific clinical markers are limited, so this approach is relatively novel for this subtype.
Where this research is happening
-
Phoenix Nest, INC.
Brooklyn, United States Active
Researchers
- Principal investigator: Singamsetty, Srikanth — Phoenix Nest, INC.
- Study coordinator: Singamsetty, Srikanth
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.