Developing a platform to predict heart disease risk in boys with Duchenne muscular dystrophy
Official title: Prospective Development of a Multimodal Biomaker Platform for Predictive Risk Stratification of Cardiac Disease in Duchenne Muscular Dystrophy
Funded at Vanderbilt University Medical Center · NIH-10795634 · Data updated 2026-11-12
This study is looking at how heart disease develops in boys with Duchenne muscular dystrophy (DMD) to help figure out who might be at greater risk for heart problems, so that doctors can provide better treatments and care.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Vanderbilt University Medical Center NIH-funded |
| Lab location | 1 site (Nashville, United States) |
| Project start | September 15, 2019 |
| Project end | February 28, 2026 |
| Funding year | 2024 |
| Award (this year) | $675,698 |
| NIH project number | 5R01FD006649-04 |
| Project ID | NIH-10795634 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are boys diagnosed with Duchenne muscular dystrophy who are at risk for developing cardiomyopathy.
✕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 improving the understanding of heart disease progression in boys with Duchenne muscular dystrophy (DMD), a severe genetic condition. By evaluating blood and imaging markers, the study aims to identify which patients are at higher risk for developing cardiomyopathy, a leading cause of death in DMD. The research will involve a large group of DMD patients and will utilize advanced imaging techniques and genetic analysis to create a comprehensive risk assessment tool. This could help in tailoring treatments and improving clinical trial designs for new therapies.
Why it matters
Potential benefit: If successful, this research could lead to better risk stratification and targeted therapies for heart disease in patients with Duchenne muscular dystrophy.
How similar studies have gone: Similar research has shown promise in using biomarkers for risk assessment in other cardiac conditions, but this specific approach in DMD is relatively novel.
Where this research is happening
-
Vanderbilt University Medical Center
Nashville, United States Active
Researchers
- Principal investigator: Soslow, Jonathan Harvey — Vanderbilt University Medical Center
- Study coordinator: Soslow, Jonathan Harvey
Related research
Other NIH-funded projects on the same topics.
-
Advancing manufacturing and release testing for a Duchenne gene editing therapy
A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.
-
Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.
-
Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy
Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.
-
MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy
This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.
-
Digital motor-skill tracking for infants with Duchenne Muscular Dystrophy using wearables
A wearable-sensor project aims to quietly track and quantify how motor skills develop in babies and young boys with Duchenne Muscular Dystrophy.
-
Genetic factors that influence Duchenne Muscular Dystrophy progression
A study to find genes that change how severe Duchenne muscular dystrophy is in boys and young men, using patient data and DNA samples.
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.