Targeting p38 MAPK to treat Friedreich ataxia
Official title: p38 MAPK activation as a therapeutic target for Friedreich ataxia
Funded at Children's Hosp of Philadelphia · NIH-11049076 · Data updated 2026-11-12
This study is looking at how a specific pathway in the body might be making Friedreich ataxia worse, and it hopes to find new treatments that could help improve symptoms and slow down the disease for those living with it.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | June 15, 2022 |
| Project end | March 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $556,671 |
| NIH project number | 5R01NS124640-04 |
| Project ID | NIH-11049076 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Friedreich ataxia, particularly those experiencing neurological and cardiac symptoms.
✕Less likely to help
Patients with other forms of ataxia or those without a diagnosis of Friedreich ataxia may not benefit from this research.
What this research studies
This research investigates the role of the p38 MAP kinase pathway in Friedreich ataxia, a genetic disorder that affects the nervous and cardiovascular systems. The study aims to understand how the hyperactivation of this pathway contributes to the disease and explores potential therapies that could inhibit this activation. By targeting the p38 pathway, the researchers hope to improve the biogenesis of iron-sulfur clusters, which are crucial for cellular function. Patients may benefit from new treatment strategies that could alleviate symptoms and slow disease progression.
Why it matters
Potential benefit: If successful, this research could lead to effective therapies for Friedreich ataxia, improving quality of life for patients.
How similar studies have gone: While targeting the p38 MAPK pathway is a novel approach in Friedreich ataxia, similar strategies have shown promise in other neurodegenerative conditions.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Wilson, Robert B — Children's Hosp of Philadelphia
- Study coordinator: Wilson, Robert B
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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.