Identifying biomarkers and clinical endpoints for myotonic dystrophy type-1
Official title: Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type-1 (Renewal)
Funded at Virginia Commonwealth University · NIH-11058441 · Data updated 2026-11-12
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 start | September 15, 2022 |
| Project end | February 28, 2027 |
| Funding year | 2025 |
| Award (this year) | $398,302 |
| NIH project number | 5R01FD006071-05 |
| Project ID | NIH-11058441 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 those without a diagnosis of myotonic dystrophy type-1 may not benefit from this research.
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.
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 gone: 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
-
Virginia Commonwealth University
Richmond, United States Active
Researchers
- Principal investigator: Johnson, Nicholas Elwood — Virginia Commonwealth University
- Study coordinator: Johnson, Nicholas Elwood
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.
-
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.
-
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.