Investigating RNA biomarkers in myotonic dystrophy type 1
Extracellular RNA biomarkers of myotonic dystrophy type 1
This study is looking for helpful markers in urine that can show how myotonic dystrophy type 1 is affecting your muscles, making it easier to track the disease without needing a muscle biopsy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10856448 on NIH RePORTER |
What this research studies
This research focuses on myotonic dystrophy type 1 (DM1), a genetic disorder that leads to muscle weakness and wasting. It aims to identify extracellular RNA (exRNA) biomarkers in urine that can reflect the severity of the disease, providing a less invasive alternative to muscle biopsies. By analyzing the RNA released from cells, the study seeks to establish a correlation between these biomarkers and clinical measures of muscle function. This could enable better monitoring of disease progression and response to potential treatments.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with myotonic dystrophy type 1, including both adults and children.
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 provide a non-invasive method for monitoring disease activity in patients with myotonic dystrophy type 1.
How similar studies have performed: Previous research has shown promise in using RNA biomarkers for monitoring other diseases, suggesting potential success for this novel approach in myotonic dystrophy.
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.