Pediatric Natural History of Myotonic Dystrophy Type 1
Official title: Pediatric Natural History
Funded at Virginia Commonwealth University · NIH-11423675 · Data updated 2026-11-12
A study following children and young people with this muscular disease to better understand how it progresses over time.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Virginia Commonwealth University NIH-funded |
| Lab location | 1 site (Richmond, United States) |
| Project start | September 25, 2025 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $537,473 |
| NIH project number | 5P50AR085906-02 |
| Project ID | NIH-11423675 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and young people with congenital or childhood-onset DM1 who have been or will be followed at the center.
✕Less likely to help
Patients not enrolled or lacking DM1-related symptoms may not directly benefit.
What this research studies
This project tracks the natural history of myotonic dystrophy type 1 from infancy through adulthood at a single center. Researchers will observe motor, cognitive, and biological changes over multiple years, while collecting data on RNA splicing that relates to symptoms. The goal is to map how the disease evolves at different ages so clinicians can anticipate needs and plan care. Participation may involve regular check-ins, medical assessments, and collection of samples as part of standard clinical follow-up. Results could guide future therapies and improve quality of life for patients and families.
Why it matters
Potential benefit: A clearer roadmap of how DM1 progresses could lead to earlier interventions and better-informed care plans for patients.
How similar studies have gone: Past natural history studies in CDM have identified a triphasic progression; this study extends observations to ChDM with potential novel insights.
Where this research is happening
-
Virginia Commonwealth University
Richmond, United States Active
Researchers
- Principal investigator: Johnson, Nicholas Elwood — Virginia Commonwealth University
- Study coordinator: Johnson, Nicholas Elwood
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.