Identifying RNA biomarkers in myotonic dystrophy
Official title: Prospective study in myotonic dystrophy to determine extracellular RNA biomarkers
Funded at Massachusetts General Hospital · NIH-10931559 · Data updated 2026-11-12
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 start | September 15, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $400,000 |
| NIH project number | 5U01FD008128-02 |
| Project ID | NIH-10931559 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with myotonic dystrophy type 1.
✕Less likely to help
Patients with other forms of muscular dystrophy or unrelated neurodegenerative diseases may not benefit from this research.
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.
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 gone: 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
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: Wheeler, Thurman M — Massachusetts General Hospital
- Study coordinator: Wheeler, Thurman M
Related research
Other NIH-funded projects on the same topics.
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New small molecule therapeutics to help myotonic dystrophy
A new NIH-funded project tests specially designed small molecules to lower harmful RNA activity in myotonic dystrophy and related diseases, aiming to reduce disease-driving RNA effects.
-
Mechanisms of muscle disease in myotonic dystrophy type 1
This project looks at why skeletal muscles weaken and waste away in adults with myotonic dystrophy type 1 and aims to uncover the underlying molecular causes.
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Extracellular vesicle biomarkers to monitor DM1 using urine and blood samples
A study looking for easy, noninvasive biomarkers in urine and blood to track disease activity in people with myotonic dystrophy type 1.
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Calcium Channel Blocker in DM1: Can amlodipine help myotonic dystrophy type 1
This study tests whether the calcium‑channel blocker amlodipine can improve symptoms and survival in myotonic dystrophy type 1 (DM1).
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Preparing therapies for DM1 across ages, with a focus on children
A broad, patient-focused effort to bring promising DM1 therapies to both children and adults by enriching trial readiness, biomarkers, and natural history data.
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Biological factors that drive variability in myotonic dystrophy
This grant explores why myotonic dystrophy can look very different from person to person by studying its genetic changes and how they affect muscle in adults.
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.