Specialized Center for Muscular Dystrophy (dystroglycanopathies)
Muscular Dystrophy Specialized Research Center
This center brings together lab and patient studies to find ways to restore a key muscle protein (matriglycan) and improve breathing and strength for people with dystroglycanopathy muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Iowa NIH-funded |
| Lab location | 1 site (Iowa City, United States) |
| Project ID | NIH-11131718 on NIH RePORTER |
What this research studies
Researchers use mouse models to study why breathing muscles fail and how matriglycan on α-dystroglycan affects muscle function. The team will test whether therapies already in clinical trials can restore full-length matriglycan and normalize respiratory function in preclinical models. Clinicians will follow an established group of patients with FKRP-related and non-FKRP dystroglycanopathies over time to map disease courses and identify groups with similar progression. That natural history work aims to improve routine care and help design more inclusive clinical trials for children, rare genotypes, and patients with advanced disease.
Who could benefit from this research
Good fit: People of any age diagnosed with dystroglycanopathy-type muscular dystrophy (including FKRP-related and non-FKRP genotypes), especially those with progressive limb-girdle weakness or respiratory involvement, would be ideal candidates.
Not a fit: People without dystroglycanopathy forms of muscular dystrophy or with unrelated neuromuscular conditions are unlikely to receive direct benefit from this center's studies.
Why it matters
Potential benefit: If successful, the work could lead to treatments that restore matriglycan, improve breathing and muscle strength, and enable better-designed clinical trials for people with dystroglycanopathies.
How similar studies have performed: Some early clinical trials target related pathways for certain dystroglycanopathies, but fully restoring matriglycan and improving respiratory function 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.