Center tackling muscular dystrophy from dystroglycan defects
Muscular Dystrophy Specialized Research Center
['FUNDING_OTHER'] · UNIVERSITY OF IOWA · NIH-11131722
Researchers are studying how problems with a muscle receptor called α-dystroglycan lead to weaker breathing in people with dystroglycan-related congenital and limb-girdle muscular dystrophy.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF IOWA (nih funded) |
| Locations | 1 site (IOWA CITY, UNITED STATES) |
| Trial ID | NIH-11131722 on ClinicalTrials.gov |
What this research studies
You would hear that the team focuses on a muscle receptor (α-dystroglycan) and a sugar chain called matriglycan that helps muscles attach to their surroundings. They study how genetic mutations change matriglycan and make respiratory muscles weak by using lab experiments, animal models, and analysis of patient tissue and samples. The researchers aim to map which genes and enzyme complexes are needed in breathing muscles and how their loss causes breathing problems. Their lab findings are meant to point toward ways to protect or restore breathing function.
Who could benefit from this research
Good fit: Ideal candidates are people with congenital or limb-girdle muscular dystrophy linked to dystroglycan pathway gene mutations, especially those with breathing weakness or declining respiratory function.
Not a fit: People whose muscle disease is caused by unrelated genes or who have no respiratory involvement may not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to new ways to preserve or improve breathing in people with dystroglycan-related muscular dystrophy.
How similar studies have performed: Genetic studies have identified many enzymes in the pathway, but applying that knowledge to respiratory muscle function and turning it into treatments remains largely experimental.
Where this research is happening
IOWA CITY, UNITED STATES
- UNIVERSITY OF IOWA — IOWA CITY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CAMPBELL, KEVIN P. — UNIVERSITY OF IOWA
- Study coordinator: CAMPBELL, KEVIN P.
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.