Exploring new treatments for muscular dystrophy
Official title: MDSRC for Next Directions in Muscular Dystrophy Research
Funded at Research Inst Nationwide Children's Hosp · NIH-10992522 · Data updated 2026-11-12
This study is exploring new gene therapies for Limb Girdle Muscular Dystrophy 2I to help improve muscle function and slow down the disease, with the hope that patients will experience better treatments and an improved quality of life.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project start | August 15, 2024 |
| Project end | July 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $1,557,819 |
| NIH project number | 1P50HD117373-01A1 |
| Project ID | NIH-10992522 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Limb Girdle Muscular Dystrophy 2I or other forms of muscular dystrophy.
✕Less likely to help
Patients with muscular dystrophy types not targeted by this research may not receive any benefit.
What this research studies
This research focuses on developing innovative gene therapies for muscular dystrophy, particularly targeting Limb Girdle Muscular Dystrophy 2I. The team at Nationwide Children's Hospital is investigating the use of advanced AAV vectors to enhance muscle function and prevent disease progression. They aim to identify new biomarkers and improve gene therapy effectiveness while minimizing immune responses. Patients may benefit from cutting-edge treatments that could significantly improve their quality of life.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking therapies that restore muscle function and slow disease progression in muscular dystrophy patients.
How similar studies have gone: Previous research has shown promise in gene therapy approaches for muscular dystrophy, indicating potential for success in this novel investigation.
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
Related research
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This grant studies how fat that builds up inside muscles in Duchenne and Becker muscular dystrophy may release hormones that affect overall health, especially metabolism.
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Stopping steroid-related muscle and bone wasting in Duchenne muscular dystrophy
Researchers are testing ways to protect muscles and bones in people with Duchenne muscular dystrophy who take long-term steroid medicines.
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Curation of muscular dystrophy genes to improve diagnosis and care
A grant to help doctors better interpret genetic tests for muscular dystrophy and myopathy so patients get clearer diagnoses and faster access to therapies.
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MR clues to how nNOS affects energy use and blood flow in Becker and Duchenne muscular dystrophy
This project looks at how advanced MRI tests can track muscle energy and blood flow changes in people with Becker and Duchenne muscular dystrophy as new gene therapies are explored.
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.