New small molecules to help treat myotonic dystrophy
Official title: Development of novel small molecules to treat myotonic dystrophy
Funded at Repeat RNA Therapeutics INC · NIH-11315150 · Data updated 2026-11-12
A new NIH grant is developing small molecule drugs to help people with myotonic dystrophy feel and function better.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Repeat RNA Therapeutics INC NIH-funded |
| Lab location | 1 site (Menands, United States) |
| Project start | September 10, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $322,177 |
| NIH project number | 1R41NS143535-01A1 |
| Project ID | NIH-11315150 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with myotonic dystrophy or related muscular dystrophy features who could participate in early drug testing and future trials.
✕Less likely to help
Individuals without DM or who cannot participate in early-stage drug testing may not benefit.
What this research studies
This project aims to create novel small molecules that counteract the disease process in myotonic dystrophy (DM), a condition that weakens muscles and can affect the heart and thinking. Researchers will focus on how these molecules interact with the cellular mechanisms that go wrong in DM, aiming to restore normal RNA splicing and muscle function. The work builds on understanding toxic RNA repeats that trap proteins and drive disease symptoms, seeking a therapy that works at the muscle level with better delivery. If successful, this approach could supplement or replace current strategies that require high doses of other treatments.
Why it matters
Potential benefit: If successful, patients with myotonic dystrophy could experience improved muscle strength and daily functioning.
How similar studies have gone: Previous approaches with RNA-targeted therapies have shown promise in related DM mechanisms, but require improvements in delivery and dosing.
Where this research is happening
-
Repeat RNA Therapeutics INC
Menands, United States Active
Researchers
- Principal investigator: Berglund, Andrew — Repeat RNA Therapeutics INC
- Study coordinator: Berglund, Andrew
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.
-
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).
-
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.
-
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.