Transformative therapies for myotonic dystrophy
Official title: Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center
Funded at University of Florida · NIH-10992312 · Data updated 2026-11-12
This study is working on new treatments for myotonic dystrophy types 1 and 2 by teaming up scientists and doctors to better understand the disease and help improve care for patients like you.
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) | $1,472,597 |
| NIH project number | 1P50NS132955-01A1 |
| Project ID | NIH-10992312 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals aged 21 and older who have been diagnosed with myotonic dystrophy type 1 or type 2.
✕Less likely to help
Patients with other forms of muscular dystrophy or those under 21 years old may not benefit from this research.
What this research studies
This research focuses on developing innovative treatments for myotonic dystrophy type 1 (DM1) and type 2 (DM2) through a collaborative effort between basic scientists and clinical researchers. The Wellstone Center will utilize advanced mouse models to study the disease mechanisms and support clinical trials aimed at improving patient outcomes. By investigating the genetic and molecular characteristics of DM1 and DM2, the research aims to accelerate the transition from laboratory findings to effective therapies for patients.
Why it matters
Potential benefit: If successful, this research could lead to groundbreaking therapies that significantly improve the quality of life for individuals affected by myotonic dystrophy.
How similar studies have gone: Previous research has shown promising results in developing therapies for myotonic dystrophy, indicating that this approach has the potential for success.
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
Related research
Other NIH-funded projects on the same topics.
-
Advancing manufacturing and release testing for a Duchenne gene editing therapy
A project to optimize how a dual-vector AAV gene editing treatment for Duchenne muscular dystrophy is made and tested, so it can move toward clinical use.
-
Understanding why Duchenne Muscular Dystrophy worsens and how to stop it
A research team at USC is studying Duchenne muscular dystrophy to find out why the disease continues to progress and how to interrupt harmful changes in muscle tissue.
-
Endocrine role of intramuscular fat in muscular dystrophy
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.
-
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.
-
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.
-
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.