Understanding how dystrophin protein affects muscle function in muscular dystrophy
Official title: Molecular Mechanisms of Dystrophin Expression in Ameliorated Phenotypes
Funded at Research Inst Nationwide Children's Hosp · NIH-11010866 · Data updated 2026-11-12
This study is looking at how the levels of a protein called dystrophin affect muscle function in people with Becker and Duchenne muscular dystrophy, and it aims to find ways to improve treatments by understanding how different therapies can boost dystrophin levels.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | January 18, 2023 |
| Project end | December 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $400,630 |
| NIH project number | 5R01NS126352-03 |
| Project ID | NIH-11010866 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Becker or Duchenne muscular dystrophy, particularly those with varying levels of dystrophin expression.
✕Less likely to help
Patients with other forms of muscular dystrophy or those without a diagnosis of dystrophinopathies may not benefit from this research.
What this research studies
This research investigates the molecular mechanisms behind dystrophin expression in patients with Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD). By examining how different types and amounts of dystrophin protein influence patient outcomes, the study aims to clarify the relationship between dystrophin levels and muscle function. The research will involve analyzing patient samples and exploring the effects of therapies designed to enhance dystrophin expression. Ultimately, the goal is to provide insights that could improve treatment strategies for these conditions.
Why it matters
Potential benefit: If successful, this research could lead to more effective therapies for muscular dystrophy, improving muscle function and quality of life for patients.
How similar studies have gone: Previous research has shown promise in understanding dystrophin's role in muscular dystrophies, suggesting that this approach could yield valuable insights.
Where this research is happening
-
Research Inst Nationwide Children's Hosp
Columbus, United States Active
Researchers
- Principal investigator: Flanigan, Kevin M — Research Inst Nationwide Children's Hosp
- Study coordinator: Flanigan, Kevin M
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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.