Fumarate drugs improve heart function in mice with Friedreich's ataxia
Fumarate drugs rescue cardiac dysfunction in mouse models of Friedreich's ataxia
This study is looking at how a drug called dimethyl fumarate might help improve heart function in mice with Friedreich's ataxia, a genetic condition that can cause serious heart problems, with hopes that it could lead to better treatments for people with the same condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California at Davis NIH-funded |
| Lab location | 1 site (Davis, United States) |
| Project ID | NIH-10975149 on NIH RePORTER |
What this research studies
This research investigates the effects of fumarate drugs on heart function in mouse models of Friedreich's ataxia, a genetic disorder that leads to severe cardiac issues. The study focuses on the drug dimethyl fumarate, which has shown promise in protecting heart cells and improving cardiac function by restoring critical mitochondrial proteins. By screening a library of existing drugs, researchers aim to find effective treatments that could potentially be translated to human patients suffering from this condition. The approach involves assessing heart performance and cellular health in mice that mimic the human disease.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Friedreich's ataxia, particularly those experiencing cardiac dysfunction.
Not a fit: Patients with Friedreich's ataxia who do not exhibit cardiac issues may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic options for patients with Friedreich's ataxia, potentially improving heart health and overall quality of life.
How similar studies have performed: Previous research has shown that similar approaches using existing drugs can yield promising results in treating related conditions, suggesting potential for success in this study.
Where this research is happening
Davis, United States
- University of California at Davis — Davis, United States (Active)
Researchers
- Principal investigator: Dedkova, Elena N. — University of California at Davis
- Study coordinator: Dedkova, Elena N.
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.