New treatments for Pompe disease
Official title: Novel Adjunctive Therapies for Pompe Disease
Funded at Duke University · NIH-11039977 · Data updated 2026-11-12
This study is looking for new ways to help people with Pompe disease breathe better and improve their muscle function by finding treatments that work alongside the current enzyme replacement therapy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | February 19, 2024 |
| Project end | January 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $249,000 |
| NIH project number | 5R00HL161420-04 |
| Project ID | NIH-11039977 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Pompe disease, particularly those experiencing respiratory issues.
✕Less likely to help
Patients with Pompe disease who do not have respiratory complications may not benefit from this research.
What this research studies
This research focuses on developing novel adjunctive therapies for Pompe disease, a serious condition caused by a deficiency in the enzyme acid α-glucosidase. The goal is to enhance existing enzyme replacement therapy by finding ways to improve the function of respiratory muscles and motor neurons affected by the disease. Researchers at Duke University will explore innovative approaches to clear glycogen buildup and repair cellular processes that are disrupted in patients. This work aims to provide better outcomes for individuals suffering from respiratory complications associated with Pompe disease.
Why it matters
Potential benefit: If successful, this research could lead to improved respiratory function and quality of life for patients with Pompe disease.
How similar studies have gone: Previous research has shown promise in gene therapy approaches for neuromuscular diseases, indicating potential for success in this novel treatment strategy.
Where this research is happening
-
Duke University
Durham, United States Active
Researchers
- Principal investigator: Roger, Angela L. — Duke University
- Study coordinator: Roger, Angela L.
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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.