Treating and understanding peripheral nerve problems in Sanfilippo (MPS IIIA)
Characterizing and treating peripheral nervous system dysfunction in Sanfilippo Syndrome
Researchers aim to find why peripheral nerves are damaged and how to treat those nerve problems in children with Sanfilippo syndrome (MPS IIIA).
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Georgia NIH-funded |
| Lab location | 1 site (Athens, United States) |
| Project ID | NIH-11264950 on NIH RePORTER |
What this research studies
This project will look for why your child's peripheral nerves are damaged in Sanfilippo and test ways to help them. The team will use mouse models and disease samples to measure harmful buildup of heparan sulfate and study nerves that control hearing, digestion, and heart function. They will test approaches designed to reach peripheral nerves and monitor whether nerve function and organ symptoms improve. If promising, the results could point toward new treatments to ease daily symptoms for children with MPS IIIA.
Who could benefit from this research
Good fit: Children diagnosed with Sanfilippo syndrome type A (MPS IIIA), particularly those experiencing sensory, autonomic, hearing, bowel, or cardiac symptoms.
Not a fit: People without MPS IIIA or patients whose problems are driven only by central nervous system damage may not directly benefit from peripheral-focused approaches.
Why it matters
Potential benefit: Could lead to treatments that reduce sensory and autonomic problems such as hearing loss, bowel and heart issues, and improve quality of life for children with MPS IIIA.
How similar studies have performed: Most prior research has focused on the brain rather than peripheral nerves, so this peripheral-focused approach is relatively novel although related gene- or enzyme-based strategies for MPS disorders have had limited success.
Where this research is happening
Athens, United States
- University of Georgia — Athens, United States (Active)
Researchers
- Principal investigator: Weiss, Ryan Joseph — University of Georgia
- Study coordinator: Weiss, Ryan Joseph
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 below.