Predicting heart disease progression in Duchenne and Becker muscular dystrophy
Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials
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 ID | NIH-11252272 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: 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.
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 performed: 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
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.