Understanding the causes and treatments for myotonic dystrophy type 2.
Official title: DM2 pathomechansims and therapeutic development
Funded at University of Florida · NIH-10992316 · Data updated 2026-11-12
This study is looking at myotonic dystrophy type 2, a genetic muscle disorder, by creating mouse models to learn more about how the disease works and to find new ways to help people with this condition since there aren't many good treatments available right now.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Florida NIH-funded |
| Lab location | 1 site (Gainesville, United States) |
| Project start | August 15, 2024 |
| Project end | July 31, 2028 |
| Funding year | 2024 |
| Award (this year) | $364,709 |
| NIH project number | 1P50NS132955-01A1 |
| Project ID | NIH-10992316 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with myotonic dystrophy type 2, particularly those experiencing muscle weakness and related symptoms.
✕Less likely to help
Patients with other forms of muscular dystrophy or those without a diagnosis of myotonic dystrophy type 2 may not benefit from this research.
What this research studies
This research focuses on myotonic dystrophy type 2 (DM2), a genetic muscular disorder caused by specific mutations in the CNBP gene. The team is developing mouse models that closely mimic the clinical features of DM2 to better understand the disease mechanisms and test potential therapies. By studying how the mutated genes affect RNA processing and muscle function, the researchers aim to identify new treatment strategies that could improve patient outcomes. This work is crucial as effective therapies for DM2 are currently lacking.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve the quality of life for individuals with myotonic dystrophy type 2.
How similar studies have gone: While research on myotonic dystrophy type 1 has shown promise, the specific approaches for DM2 are relatively novel and have not been extensively tested.
Where this research is happening
-
University of Florida
Gainesville, United States Active
Researchers
- Principal investigator: Swanson, Maurice Scott — University of Florida
- Study coordinator: Swanson, Maurice Scott
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.