Extracellular Vesicles to Monitor and Improve Dystrophinopathy
Official title: (Project 3) Extracellular Vesicles in Monitoring and Treatment of Dystrophinopathy
Funded at Research Inst Nationwide Children's Hosp · NIH-11397458 · Data updated 2026-11-12
A patient-friendly grant focusing on Duchenne muscular dystrophy uses tiny particles in the blood to track disease and help guide future therapies.
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 | 2026 |
| Award (this year) | $299,773 |
| NIH project number | 5P50HD117373-03 |
| Project ID | NIH-11397458 on NIH RePORTER |
Who could benefit from this research
✓Could help
Boys with Duchenne muscular dystrophy, particularly around adolescence, who are part of or eligible for gene therapy trials.
✕Less likely to help
Patients not enrolled in DMD-related trials or without access to gene therapy studies may see limited direct benefit.
What this research studies
This project studies extracellular vesicles to non-invasively monitor disease activity in boys with Duchenne muscular dystrophy and to support the development of better gene therapies. It aims to link biomarker signals from vesicles with clinical outcomes to understand how well treatments work over time. The work involves collaboration with a national children’s hospital to study real patients and their treatment responses. Results could lead to easier monitoring and safer, more effective therapies for DMD.
Why it matters
Potential benefit: If successful, this research could improve how doctors track DMD progression and tailor therapies to individual patients.
How similar studies have gone: Similar non-invasive monitoring approaches have shown promise in other muscular dystrophies, but this approach remains exploratory for DMD.
Where this research is happening
-
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.