Small-molecule treatments for myotonic dystrophy type 1
Official title: Small molecule therapeutics for myotonic dystrophy type 1
Funded at University of Southern California · NIH-11263628 · Data updated 2026-11-12
Developing new small-molecule drugs that target the toxic RNA that causes myotonic dystrophy type 1 for people living with DM1.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Southern California NIH-funded |
| Lab location | 1 site (Los Angeles, UNITED STATES) |
| Project start | January 15, 2023 |
| Project end | December 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $515,087 |
| NIH project number | 5R01NS126219-04 |
| Project ID | NIH-11263628 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with genetically confirmed myotonic dystrophy type 1 (expanded CTG repeats in DMPK) who are interested in future clinical trials would be the likely candidates.
✕Less likely to help
Individuals without DM1, or those whose symptoms are due to other conditions, would not benefit from this DM1-specific research.
What this research studies
This project looks for drug-like small molecules that bind the expanded CUG repeat RNA from the DMPK gene, the toxic RNA that drives DM1. The team used an RNA-based screen of 40,000 compounds and found multiple hits that reduce nuclear RNA foci in patient cells and perform well compared with a prototype compound (MDI16) and antisense oligonucleotides. Leads are being rank-ordered by potency, selectivity, efficacy, and safety in cellular assays and in the HSALR mouse model. The best candidates would be advanced toward further preclinical development and potential future human testing.
Why it matters
Potential benefit: If successful, this work could produce drug candidates that reduce toxic RNA and improve muscle and neurological symptoms in people with DM1.
How similar studies have gone: Related antisense oligonucleotide approaches and the prototype small molecule MDI16 have improved molecular and tissue features of DM1 in patient cells and mouse models, though clear benefit in people has not yet been established.
Where this research is happening
-
University of Southern California
Los Angeles, United States Active
Researchers
- Principal investigator: Comai, Lucio — University of Southern California
- Study coordinator: Comai, Lucio
Related research
Other NIH-funded projects on the same topics.
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New small molecule therapeutics to help myotonic dystrophy
A new NIH-funded project tests specially designed small molecules to lower harmful RNA activity in myotonic dystrophy and related diseases, aiming to reduce disease-driving RNA effects.
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Mechanisms of muscle disease in myotonic dystrophy type 1
This project looks at why skeletal muscles weaken and waste away in adults with myotonic dystrophy type 1 and aims to uncover the underlying molecular causes.
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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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Calcium Channel Blocker in DM1: Can amlodipine help myotonic dystrophy type 1
This study tests whether the calcium‑channel blocker amlodipine can improve symptoms and survival in myotonic dystrophy type 1 (DM1).
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Preparing therapies for DM1 across ages, with a focus on children
A broad, patient-focused effort to bring promising DM1 therapies to both children and adults by enriching trial readiness, biomarkers, and natural history data.
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Biological factors that drive variability in myotonic dystrophy
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.
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.