Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy
Official title: Targeting Glucocorticoid Atrophy Signaling to Treat Duchenne Muscular Dystrophy
Funded at Univ of Arkansas for Med Scis · NIH-11458914 · Data updated 2026-11-12
Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Arkansas for Med Scis NIH-funded |
| Lab location | 1 site (Little Rock, United States) |
| Project start | February 16, 2018 |
| Project end | January 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $261,556 |
| NIH project number | 5P20GM125503-09 |
| Project ID | NIH-11458914 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with Duchenne muscular dystrophy—especially those on long-term glucocorticoid (steroid) therapy—are the population this research is intended to help.
✕Less likely to help
People without DMD, or whose muscle or bone problems come from other causes, are unlikely to benefit directly from this research.
What this research studies
This project looks at how the steroid medicines commonly used for Duchenne muscular dystrophy cause muscle and bone loss and tests ways to block those harmful signals. The team focuses on molecules called Atrogin1 and MuRF1 that tag proteins for destruction and uses lab-grown cells, genetic approaches, and mouse models plus drugs that inhibit protein degradation. Early results showed that blocking these signals or using a proteasome inhibitor helped muscle function and bone density in mice, and the project will extend that work toward strategies that could protect people taking glucocorticoids.
Why it matters
Potential benefit: If successful, the work could lead to treatments that prevent steroid-related muscle and bone loss in people with DMD, improving strength and bone health.
How similar studies have gone: Preclinical studies in cells and mice have shown that blocking atrophy genes like MuRF1 or using proteasome inhibitors can protect muscle and bone, but these approaches have not yet been proven safe or effective in people.
Where this research is happening
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Univ of Arkansas for Med Scis
Little Rock, United States Active
Researchers
- Principal investigator: Sato, Amy Yoshiko — Univ of Arkansas for Med Scis
- Study coordinator: Sato, Amy Yoshiko
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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.