Investigating treatments for a rare childhood brain disorder called MPS IIID.
Official title: Evaluation of Clinical Outcome Assessment (COA) and Potential Biomarkers to Facilitate Interventional Trial for Mucopolysaccharidosis IIID Patients
Funded at Phoenix Nest, INC. · NIH-10893861 · Data updated 2026-11-12
This study is looking at a new treatment for Mucopolysaccharidosis IIID, a rare condition that affects how children's brains develop, and it aims to help improve their quality of life by delivering a special enzyme directly into the brain.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Phoenix Nest, INC. NIH-funded |
| Lab location | 1 site (Brooklyn, United States) |
| Project start | September 21, 2021 |
| Project end | August 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $100,000 |
| NIH project number | 3UB1NS122644-04S1 |
| Project ID | NIH-10893861 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with MPS IIID, particularly those in the early stages of the disease.
✕Less likely to help
Patients with other forms of Mucopolysaccharidosis or those who are not diagnosed with MPS IIID may not benefit from this research.
What this research studies
This research focuses on Mucopolysaccharidosis IIID (MPS IIID), a rare neurodegenerative disorder that affects children's brain development and leads to severe cognitive and motor decline. The team is exploring a potential enzyme replacement therapy (ERT) that delivers a specific enzyme directly into the brain to address the underlying causes of the disease. By using intracerebroventricular infusion, they aim to improve the quality of life and developmental outcomes for affected children. The research is particularly significant as there are currently no available treatments for this condition.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that significantly improves the quality of life for children with MPS IIID.
How similar studies have gone: There have been successful examples of enzyme replacement therapies for other forms of Mucopolysaccharidosis, indicating potential for success with this novel approach.
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.