Genetic factors that influence Duchenne Muscular Dystrophy progression
Official title: Genetic modifiers of Duchenne Muscular Dystrophy
Funded at Research Inst Nationwide Children's Hosp · NIH-11377465 · Data updated 2026-11-12
A study to find genes that change how severe Duchenne muscular dystrophy is in boys and young men, using patient data and DNA samples.
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 | May 15, 2014 |
| Project end | July 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $921,491 |
| NIH project number | 5R01NS085238-10 |
| Project ID | NIH-11377465 on NIH RePORTER |
Who could benefit from this research
✓Could help
Young men and boys with Duchenne muscular dystrophy or related dystrophinopathies who are represented in the UDP database.
✕Less likely to help
Patients whose disease is not influenced by the identified modifiers or who are not represented in the UDP dataset may see no direct benefit.
What this research studies
This project looks at genetic modifiers that may affect how quickly Duchenne Muscular Dystrophy progresses in skeletal muscles, the heart, and breathing. Researchers will analyze phenotypic data and archived DNA from the United Dystrophinopathy Project to identify genes linked to milder or more severe disease courses. They will update the UDP dataset and map traits related to disease progression using existing patient information to find potential modifier genes. The goal is to better understand why some patients fare differently and to inform future therapies. Results could guide personalized approaches to care and treatment decisions for individuals with DMD.
Why it matters
Potential benefit: Identifying modifier genes could lead to new targets for therapies and more personalized care plans for people with DMD.
How similar studies have gone: Prior work has shown that specific gene variants (like LTBP4) can influence disease progression in DMD, supporting the potential for new modifiers to be found.
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.