Developing a platform to predict heart disease risk in boys with Duchenne muscular dystrophy
Prospective Development of a Multimodal Biomaker Platform for Predictive Risk Stratification of Cardiac Disease in Duchenne Muscular Dystrophy
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 ID | NIH-10795634 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are boys diagnosed with Duchenne muscular dystrophy who are at risk for developing cardiomyopathy.
Not a fit: Patients with other forms of muscular dystrophy or those without a diagnosis of Duchenne muscular dystrophy may not benefit from this research.
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 performed: 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
Nashville, United States
- Vanderbilt University Medical Center — Nashville, United States (Active)
Researchers
- Principal investigator: Soslow, Jonathan Harvey — Vanderbilt University Medical Center
- Study coordinator: Soslow, Jonathan Harvey
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 below.