Identifying RNA biomarkers in myotonic dystrophy
Prospective study in myotonic dystrophy to determine extracellular RNA biomarkers
This study is looking at myotonic dystrophy to see if certain RNA patterns in spinal fluid can help us understand how the disease is changing over time and how well treatments are working, which could lead to better ways to diagnose and treat patients like you.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10931559 on NIH RePORTER |
What this research studies
This research focuses on myotonic dystrophy, a genetic condition that leads to muscle weakness and cognitive issues. The team aims to explore the role of extracellular RNA as potential biomarkers that could indicate disease progression or response to treatment. By analyzing cerebrospinal fluid, they hope to identify specific RNA patterns that correlate with the disease's effects on the brain and muscles. This could pave the way for new diagnostic tools and therapeutic strategies for patients.
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 unrelated neurodegenerative diseases may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to earlier diagnosis and better monitoring of myotonic dystrophy, ultimately improving patient care.
How similar studies have performed: While the use of extracellular RNA as biomarkers is a growing field, this specific approach in myotonic dystrophy is relatively novel and has not been extensively tested.
Where this research is happening
Boston, United States
- Massachusetts General Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Wheeler, Thurman M — Massachusetts General Hospital
- Study coordinator: Wheeler, Thurman 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 below.