Predicting heart disease progression in Duchenne and Becker muscular dystrophy
Official title: Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials
Funded at Vanderbilt University Medical Center · NIH-11252272 · Data updated 2026-11-12
This project will look for blood tests and heart-imaging signs that predict how heart disease will progress in people with Duchenne or Becker muscular dystrophy and in genetic carriers.
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 | January 23, 2024 |
| Project end | December 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $699,395 |
| NIH project number | 5R01HL167969-03 |
| Project ID | NIH-11252272 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are people with genetically confirmed Duchenne or Becker muscular dystrophy and confirmed dystrophin mutation carriers who can share medical records and attend periodic cardiac MRI visits.
✕Less likely to help
People without DMD/BMD or dystrophin mutations, those unable to undergo cardiac MRI (for example due to incompatible implants or severe claustrophobia), or those who cannot access a participating center are unlikely to benefit directly.
What this research studies
You would be invited to join a prospective registry that collects clinical information, blood samples, and regular cardiac MRI (CMR) scans from people with DMD, BMD, and mutation carriers. The project pools data from eight high-volume centers using similar imaging and care protocols, aiming to enroll about 950 participants and analyze over 4,000 CMR studies. Researchers will compare imaging features, clinical measurements, and blood markers to find signals that predict rapid heart decline or early death. The goal is to create reliable surrogate outcome measures that make future cardiomyopathy treatment trials faster and more informative.
Why it matters
Potential benefit: If successful, this work could identify people at higher risk of severe heart problems and provide validated outcome measures that speed up and improve clinical trials for DMD/BMD heart disease.
How similar studies have gone: Smaller studies have shown cardiac MRI can detect heart damage in DMD/BMD, but using those measures as validated surrogate endpoints for trials remains largely unproven and this large, standardized registry is a novel effort.
Where this research is happening
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Vanderbilt University Medical Center
Nashville, United States Active
Researchers
- Principal investigator: Soslow, Jonathan Harvey — Vanderbilt University Medical Center
- Study coordinator: Soslow, Jonathan Harvey
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This study is looking at how a protein called Connexin 43 affects heart health in people with Duchenne muscular dystrophy, with the goal of finding new ways to help prevent heart problems like arrhythmias and heart failure.
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Understanding heart problems in Duchenne and Becker muscular dystrophy
This study is looking at heart problems that can happen in people with Duchenne and Becker muscular dystrophy to find better ways to check heart health and track how the disease changes over time, which will help improve treatments and future drug trials for those affected.
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New treatment for heart failure in Duchenne muscular dystrophy patients
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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.