Developing therapies for Duchenne muscular dystrophy and related conditions
Official title: MDSRC Administrative Core
Funded at Research Inst Nationwide Children's Hosp · NIH-10992523 · Data updated 2026-11-12
This study is looking at new gene therapy treatments for Duchenne muscular dystrophy (DMD) to help improve muscle function and slow down the disease, and it's designed for families and individuals affected by DMD who are hoping for better options.
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) | $145,228 |
| NIH project number | 1P50HD117373-01A1 |
| Project ID | NIH-10992523 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with Duchenne muscular dystrophy or related muscular dystrophies, particularly those who may benefit from gene therapy approaches.
✕Less likely to help
Patients with muscular dystrophies not targeted by the specific therapies being developed, or those who do not meet the eligibility criteria for participation, may not receive benefit from this research.
What this research studies
This research focuses on advancing therapies for Duchenne muscular dystrophy (DMD) and other muscular dystrophies through innovative gene therapy techniques. The team at the Abigail Wexner Research Institute collaborates with experts to explore new treatment paradigms, including the use of AAV vectors to enhance muscle function and prevent disease progression. By sharing their expertise with the broader muscular dystrophy research community, they aim to translate laboratory findings into effective clinical treatments. The research includes projects that investigate novel gene delivery methods and identify biomarkers for disease progression.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking therapies that significantly improve muscle function and quality of life for patients with Duchenne muscular dystrophy.
How similar studies have gone: Previous research has shown promise in using gene therapy approaches for muscular dystrophies, indicating that this line of investigation has the potential for significant advancements.
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
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.