Developing new small molecule treatments for myotonic dystrophy
Official title: Design, Synthesis and Efficacy of New Small Molecule Therapeutics to Impede Myotonic Dystrophy
Funded at State University of New York at Albany · NIH-11060026 · Data updated 2026-11-12
This study is working on new medicines to help people with myotonic dystrophy by creating special compounds that can target and reduce the harmful RNA causing the condition, with the hope of easing symptoms and improving lives.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | State University of New York at Albany NIH-funded |
| Lab location | 1 site (Albany, United States) |
| Project start | May 1, 2022 |
| Project end | March 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $425,205 |
| NIH project number | 5R01NS120485-04 |
| Project ID | NIH-11060026 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with myotonic dystrophy, particularly those with the DM1 or DM2 forms of the disease.
✕Less likely to help
Patients with other forms of neuromuscular disorders unrelated to myotonic dystrophy may not benefit from this research.
What this research studies
This research focuses on creating new small molecule therapeutics aimed at treating myotonic dystrophy, a neuromuscular disorder caused by toxic RNA. The approach involves designing modified polycyclic compounds that can selectively reduce the harmful expanded RNAs responsible for the disease. By understanding the mechanisms of action of these compounds, the research aims to identify effective drug candidates that can alleviate symptoms and improve patient outcomes. The study utilizes cell lines from patients with myotonic dystrophy to test the efficacy of these new treatments.
Why it matters
Potential benefit: If successful, this research could lead to effective new therapies that significantly improve the quality of life for patients with myotonic dystrophy.
How similar studies have gone: Previous research has shown promise with similar small molecule approaches in reducing toxic RNA levels in myotonic dystrophy, indicating potential for success in this novel therapeutic strategy.
Where this research is happening
-
State University of New York at Albany
Albany, United States Active
Researchers
- Principal investigator: Berglund, Andrew — State University of New York at Albany
- 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.
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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.