Using a new drug to improve heart and muscle function in patients with Duchenne muscular dystrophy
Official title: TRPC6 inhibition therapy to rescue cardiac muscle dysfunction in muscular dystrophy
Funded at Medical College of Wisconsin · NIH-11057688 · Data updated 2026-11-12
This study is testing a new drug that might help protect the hearts and muscles of people with Duchenne muscular dystrophy by blocking a protein that causes damage, and it's being tried out in mice and heart tissues to see if it can really make a difference.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | March 18, 2024 |
| Project end | February 28, 2027 |
| Funding year | 2025 |
| Award (this year) | $249,000 |
| NIH project number | 5R00HL155840-04 |
| Project ID | NIH-11057688 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Duchenne muscular dystrophy, particularly those experiencing cardiac and skeletal muscle dysfunction.
✕Less likely to help
Patients with other forms of muscular dystrophy or those without cardiac involvement may not benefit from this research.
What this research studies
This research investigates a new small-molecule drug that inhibits a specific protein called TRPC6, which is known to contribute to heart and muscle problems in patients with Duchenne muscular dystrophy (DMD). The study aims to understand how this drug can reduce muscle damage and improve heart function by conducting experiments in mice and engineered heart tissues. If successful, this approach could lead to new treatments that significantly enhance the quality of life for individuals affected by DMD.
Why it matters
Potential benefit: If successful, this research could provide a novel therapy that improves heart and muscle function in patients with Duchenne muscular dystrophy.
How similar studies have gone: Preliminary data from similar research have shown promising results, indicating that TRPC6 inhibition can significantly improve outcomes in models of Duchenne muscular dystrophy.
Where this research is happening
-
Medical College of Wisconsin
Milwaukee, United States Active
Researchers
- Principal investigator: Lin, Brian Leei — Medical College of Wisconsin
- Study coordinator: Lin, Brian Leei
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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.