Understanding Muscle Weakness in Myotonic Dystrophy
Official title: Mechanism of Skeletal Muscle Calcium Dysregulation in Myotonic Dystrophy
Funded at University of Rochester · NIH-11101114 · Data updated 2026-11-12
This research aims to understand why muscles weaken in Myotonic Dystrophy and explore if an existing medication could help.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project start | August 8, 2022 |
| Project end | June 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $465,103 |
| NIH project number | 5R01AR079424-04 |
| Project ID | NIH-11101114 on NIH RePORTER |
Who could benefit from this research
✓Could help
This foundational research is relevant for adults aged 21 and older living with Myotonic Dystrophy.
✕Less likely to help
Patients without Myotonic Dystrophy would not directly benefit from this specific research.
What this research studies
Myotonic Dystrophy causes muscles to weaken because certain genetic instructions for muscle function get mixed up, similar to how they are in babies. This project uses specially developed mouse models that mimic these genetic changes to understand exactly how they lead to severe muscle problems and breathing difficulties. Researchers are also testing if a calcium channel blocker, a type of medication already approved for other uses, can improve muscle strength and breathing in these models. The goal is to find new ways to treat muscle weakness in people with Myotonic Dystrophy.
Why it matters
Potential benefit: If successful, this work could lead to new or repurposed drug treatments that improve muscle strength and breathing for individuals with Myotonic Dystrophy.
How similar studies have gone: Preliminary studies in mouse models have shown that an FDA-approved calcium channel blocker can rescue severe muscle weakness and respiratory issues.
Where this research is happening
-
University of Rochester
Rochester, United States Active
Researchers
- Principal investigator: Lueck, John D. — University of Rochester
- Study coordinator: Lueck, John D.
Related research
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Mechanisms of muscle disease in myotonic dystrophy type 1
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Extracellular vesicle biomarkers to monitor DM1 using urine and blood samples
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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.