Understanding Muscle Weakness in Myotonic Dystrophy
Mechanism of Skeletal Muscle Calcium Dysregulation in Myotonic Dystrophy
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 ID | NIH-11101114 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: This foundational research is relevant for adults aged 21 and older living with Myotonic Dystrophy.
Not a fit: Patients without Myotonic Dystrophy would not directly benefit from this specific research.
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 performed: 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
Rochester, United States
- University of Rochester — Rochester, United States (Active)
Researchers
- Principal investigator: Lueck, John D. — University of Rochester
- Study coordinator: Lueck, John D.
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.