Identifying biomarkers and clinical endpoints for myotonic dystrophy type-1
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type-1 (Renewal)
This study is looking at myotonic dystrophy type-1, a common muscle disease in adults, to find better ways to measure how severe the condition is and how well new treatments might work, so we can help improve care for people living with this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Virginia Commonwealth University NIH-funded |
| Lab location | 1 site (Richmond, United States) |
| Project ID | NIH-11058441 on NIH RePORTER |
What this research studies
This research focuses on myotonic dystrophy type-1 (DM1), the most common muscular dystrophy in adults, which is caused by genetic mutations leading to various symptoms such as muscle weakness and cardiac issues. The study aims to identify clinical endpoints and biomarkers that can help in assessing the severity of the disease and the effectiveness of potential therapies. By analyzing the genetic and clinical data from participants, researchers hope to improve the design of clinical trials and develop targeted treatments. The research also includes a natural history study to gather comprehensive data on the progression of DM1.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with myotonic dystrophy type-1.
Not a fit: Patients with other forms of muscular dystrophy or those without a diagnosis of myotonic dystrophy type-1 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to better-targeted therapies and improved management of myotonic dystrophy type-1.
How similar studies have performed: Previous research has shown promise in identifying biomarkers and clinical endpoints in similar muscular dystrophy conditions, indicating potential for success in this area.
Where this research is happening
Richmond, United States
- Virginia Commonwealth University — Richmond, United States (Active)
Researchers
- Principal investigator: Johnson, Nicholas Elwood — Virginia Commonwealth University
- Study coordinator: Johnson, Nicholas Elwood
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 below.