New treatments for muscle ion channel disorders
Novel Approaches to Therapy of Muscle Ion Channelopathies
['FUNDING_R01'] · WRIGHT STATE UNIVERSITY · NIH-11321140
Researchers are testing whether targeting a specific sodium current can reduce muscle stiffness and prevent weakness in people with myotonia congenita and hyperkalemic periodic paralysis.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | WRIGHT STATE UNIVERSITY (nih funded) |
| Locations | 1 site (DAYTON, UNITED STATES) |
| Trial ID | NIH-11321140 on ClinicalTrials.gov |
What this research studies
This project uses mouse models, drug testing, and computer simulations to understand why muscle cells fire too easily in myotonia congenita and become weak in hyperkalemic periodic paralysis (HPP). The team will compare the roles of the persistent sodium current (NaP) and the transient sodium current (NaT) using genetic models, selective drugs, and computerized models. They aim to find out if blocking NaP while sparing NaT can relieve stiffness without causing weakness. Results could guide development of new, more selective sodium channel medicines for patients.
Who could benefit from this research
Good fit: People with diagnosed myotonia congenita or hyperkalemic periodic paralysis, especially those whose symptoms are not well controlled by current treatments, would be the most relevant candidates for future trials.
Not a fit: Patients whose muscle symptoms are caused by non-channel disorders or unrelated conditions are unlikely to benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to new medicines that reduce myotonia and prevent paralysis attacks with fewer side effects.
How similar studies have performed: Some sodium channel blockers help myotonia but have not worked well for HPP, and selectively blocking the persistent sodium current (NaP) is a novel approach that has not yet been proven in patients.
Where this research is happening
DAYTON, UNITED STATES
- WRIGHT STATE UNIVERSITY — DAYTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: RICH, MARK M — WRIGHT STATE UNIVERSITY
- Study coordinator: RICH, MARK M
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.