Better motor tests for children with FSHD
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
This project will develop better motor measurements and trial plans to help bring treatments to children who develop FSHD early in life.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kansas Medical Center NIH-funded |
| Lab location | 1 site (Kansas City, United States) |
| Project ID | NIH-11383981 on NIH RePORTER |
What this research studies
You would be followed by specialists across the FSHD Clinical Trials Research Network who will collect motor function data and other clinical measures over time. The team will combine clinical exams, genetic details like D4Z4 repeat counts, and advanced measurements (including 3-D or imaging measures) to find which tests reliably show change in pediatric FSHD. Multiple sites will enroll and track children to improve recruitment and follow-up compared with past small studies. The chosen outcomes will be used to design future pediatric trials, including approaches that use antisense drugs or AAV-based gene therapies.
Who could benefit from this research
Good fit: Children and adolescents with genetically confirmed FSHD and symptom onset in childhood are the main candidates for this effort.
Not a fit: Adults with late-onset FSHD or people without FSHD are unlikely to get direct benefit from this pediatric-focused project.
Why it matters
Potential benefit: If successful, this work could speed up pediatric FSHD trials and make it easier to test treatments that help children sooner.
How similar studies have performed: Prior cross-sectional studies and adult treatment programs exist, but prospective pediatric outcome validation is limited and this multi-site pediatric effort is relatively novel.
Where this research is happening
Kansas City, United States
- University of Kansas Medical Center — Kansas City, United States (Active)
Researchers
- Principal investigator: Statland, Jeffrey — University of Kansas Medical Center
- Study coordinator: Statland, Jeffrey
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.