Muscular Dystrophy Specialized Research Center (P50)
Official title: Muscular Dystrophy Specialized Research Center
Funded at University of Iowa · NIH-11402658 · Data updated 2026-11-12
A long-running research program at the University of Iowa is studying the natural course of dystroglycanopathies to improve care and guide future trials for people of all ages with FKRP-related and other gene mutations.
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 start | June 8, 2005 |
| Project end | June 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $446,243 |
| NIH project number | 5P50NS053672-22 |
| Project ID | NIH-11402658 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with FKRP-related dystroglycanopathy or other dystroglycanopathy gene mutations, spanning from early childhood to late adulthood, including those previously excluded from trials.
✕Less likely to help
People not affected by dystroglycanopathies or whose condition is unrelated to the studied biomarkers and outcomes.
What this research studies
This grant supports a center-led effort to map how dystroglycanopathy conditions progress over time using standard clinical measures and new biomarkers. Researchers will follow a diverse group of over 100 participants with FKRP mutations and extend this approach to additional dystroglycanopathy genes, including children and individuals with advanced disease who often face trial-exclusion barriers. They will use remote assessments to capture real-world motor outcomes and link them to care needs like respiratory support. The work aims to identify which outcome measures best reflect disease progression and how to group patients for gene-non-specific trials. A biomarker exploration, including electroretinography, is also planned to enhance disease tracking and trial design.
Why it matters
Potential benefit: If successful, this work could clarify how dystroglycanopathies progress and improve trial design and patient care.
How similar studies have gone: Previous cohorts have shown that standardized measures can track motor progression, supporting the potential for this approach to be informative for clinical trials.
Where this research is happening
-
University of Iowa
Iowa City, United States Active
Researchers
- Principal investigator: Mathews, Katherine Dianne — University of Iowa
- Study coordinator: Mathews, Katherine Dianne
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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.