Extracellular vesicle biomarkers to monitor DM1 using urine and blood samples
Official title: Prospective natural history study of extracellular vesicle metabolimic biomarkers of myotonic dystrophy
Funded at Massachusetts General Hospital · NIH-11494099 · Data updated 2026-11-12
A study looking for easy, noninvasive biomarkers in urine and blood to track disease activity in people with myotonic dystrophy type 1.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $400,000 |
| NIH project number | 1R01FD008236-01A1 |
| Project ID | NIH-11494099 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with myotonic dystrophy type 1 who are able to participate in regular follow-up visits.
✕Less likely to help
Patients who cannot participate in serial sample collection or have contraindications to blood/urine collection may not benefit.
What this research studies
This project follows people with DM1 over time to see if molecules in extracellular vesicles found in urine and blood reflect how the disease is progressing or responding to treatment. Researchers will collect samples at multiple visits and analyze vesicle-related RNAs, proteins, and metabolites to identify reliable biomarkers. The goal is to enable frequent, painless monitoring that could reduce the need for muscle biopsies in future trials. Participation would involve scheduled visits, sample collection, and simple questions about health and symptoms. If successful, this approach could simplify tracking disease status and drug effects for patients and clinicians.
Why it matters
Potential benefit: If successful, patients could benefit from less invasive monitoring and faster assessment of treatment effects.
How similar studies have gone: Similar biomarker approaches have shown promise in other diseases, but this DM1-specific urinary EV metabolomic biomarker work is still being tested for its effectiveness.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Wheeler, Thurman M — Massachusetts General Hospital
- Study coordinator: Wheeler, Thurman M
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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.