Using extracellular vesicles to monitor and treat Duchenne Muscular Dystrophy
Official title: (Project 3) Extracellular Vesicles in Monitoring and Treatment of Dystrophinopathy
Funded at Research Inst Nationwide Children's Hosp · NIH-10992528 · Data updated 2026-11-12
This study is looking at ways to track how Duchenne Muscular Dystrophy affects boys over time and to see how well new gene therapies are working, using tiny particles from cells to make the process easier and more helpful for patients.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | August 15, 2024 |
| Project end | July 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $317,449 |
| NIH project number | 1P50HD117373-01A1 |
| Project ID | NIH-10992528 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are boys diagnosed with Duchenne Muscular Dystrophy.
✕Less likely to help
Patients with other forms of muscular dystrophy or those without a diagnosis of Duchenne Muscular Dystrophy may not benefit from this research.
What this research studies
This research focuses on Duchenne Muscular Dystrophy (DMD), a genetic condition affecting muscle function in boys. It aims to develop non-invasive methods to monitor disease progression and the effectiveness of new gene therapies. By analyzing extracellular vesicles, which are tiny particles released by cells, the researchers hope to gain insights into how well treatments are working over time. This approach could simplify the monitoring process for patients and provide valuable information for future therapies.
Why it matters
Potential benefit: If successful, this research could lead to improved monitoring of treatment effects in DMD, potentially accelerating the development of effective therapies.
How similar studies have gone: Previous research has shown promise in using similar non-invasive monitoring techniques in other conditions, suggesting potential success in this area as well.
Where this research is happening
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Research Inst Nationwide Children's Hosp
Columbus, United States Active
Researchers
- Principal investigator: Saad, Nizar — Research Inst Nationwide Children's Hosp
- Study coordinator: Saad, Nizar
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.