Preparing therapies for DM1 across ages, with a focus on children
Official title: Preparing for Therapeutics across the Spectrum of Myotonic Dystrophy
Funded at Virginia Commonwealth University · NIH-11423652 · Data updated 2026-11-12
A broad, patient-focused effort to bring promising DM1 therapies to both children and adults by enriching trial readiness, biomarkers, and natural history data.
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) | $1,281,973 |
| NIH project number | 5P50AR085906-02 |
| Project ID | NIH-11423652 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with congenital or childhood-onset DM1, and adults with DM1 may be considered for related components of the program.
✕Less likely to help
Some DM1 patients may not directly benefit if they are not enrolled in observational cohorts or if their disease course does not align with the study measures.
What this research studies
This program aims to accelerate access to treatments for myotonic dystrophy type 1 by expanding studies beyond adults to include children, collecting important clinical data and biomarkers over time. It will enroll children with congenital or childhood-onset DM1 in a two-year observational study, following their motor function, cognition, and quality of life. Researchers will also explore genetic factors that might influence disease severity and response to therapies, building a more complete picture to guide future trials. The center will leverage existing networks and prior work to harmonize data and maximize patient benefit across the community.
Why it matters
Potential benefit: If successful, patients could gain earlier access to effective therapies and more personalized treatment plans informed by richer data.
How similar studies have gone: Previous DM1 trials suggest promise for targeted therapies, but there is a need for broader age inclusion and biomarker data to optimize use.
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
Related research
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Extracellular vesicle biomarkers to monitor DM1 using urine and blood samples
A study looking for easy, noninvasive biomarkers in urine and blood to track disease activity in people with myotonic dystrophy type 1.
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This study tests whether the calcium‑channel blocker amlodipine can improve symptoms and survival in myotonic dystrophy type 1 (DM1).
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This grant explores why myotonic dystrophy can look very different from person to person by studying its genetic changes and how they affect muscle in adults.
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Identifying RNA Splicing and Gene Expression Changes in congenital myotonic dystrophy
This grant looks at how RNA changes contribute to congenital myotonic dystrophy to understand why symptoms change as children grow.
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.