Understanding Mitochondrial Myopathy and its Effects
Official title: Addressing the Research Gap in Mitochondrial Myopathy with Validated Outcome Measures and Natural History
Funded at Children's Hosp of Philadelphia · NIH-10979490 · Data updated 2026-11-12
This study is looking at Mitochondrial Myopathy, a condition that causes muscle weakness and tiredness, and aims to create better ways to track how the disease progresses, so that we can understand it more clearly and improve future treatments.
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 | August 1, 2024 |
| Project end | July 31, 2029 |
| Funding year | 2024 |
| Award (this year) | $662,241 |
| NIH project number | 1R01AR083552-01A1 |
| Project ID | NIH-10979490 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Mitochondrial Myopathy, regardless of age.
✕Less likely to help
Patients with other forms of muscle disorders that are not related to mitochondrial dysfunction may not benefit from this research.
What this research studies
This research focuses on Primary Mitochondrial Disease (PMD), specifically Mitochondrial Myopathy (MM), which causes symptoms like muscle weakness and fatigue. The study aims to develop and validate new outcome measures to better assess the progression of MM and to establish a natural history of the disease. By utilizing the MM Composite Assessment Tool (MM-COAST), researchers will gather detailed data on muscle strength, exercise tolerance, and other key areas. This comprehensive approach will help in understanding the disease better and potentially guide future treatments.
Why it matters
Potential benefit: If successful, this research could lead to improved assessment tools for Mitochondrial Myopathy, enhancing patient care and treatment options.
How similar studies have gone: Previous research has shown promise in developing outcome measures for similar genetic disorders, indicating a potential for success in this area.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Zolkipli Cunningham, Zarazuela — Children's Hosp of Philadelphia
- Study coordinator: Zolkipli Cunningham, Zarazuela
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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.