Small-molecule medicines targeting 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-11307593 · Data updated 2026-11-12
New small-molecule medicines aim to lower the toxic RNAs that cause symptoms in people with myotonic dystrophy.
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 | 2026 |
| Award (this year) | $502,351 |
| NIH project number | 5R01NS120485-05 |
| Project ID | NIH-11307593 on NIH RePORTER |
Who could benefit from this research
✓Could help
People diagnosed with myotonic dystrophy type 1 or type 2 who are willing to provide samples or consider joining future clinical testing would be the ideal candidates.
✕Less likely to help
People without myotonic dystrophy or whose symptoms arise from unrelated conditions would not be expected to benefit from this approach.
What this research studies
Researchers are designing and synthesizing modified polycyclic compounds (MPCs) that target the expanded RNAs responsible for myotonic dystrophy. They will vary the molecular core, a benzimidazole side group, and end-group chemistry to create a panel of candidate drugs. Promising compounds are screened in lab-grown cells derived from people with DM1 and DM2 to see if they restore normal RNA processing at very low concentrations. Leads will be tested further for safety and effectiveness before any clinical testing in people.
Why it matters
Potential benefit: If successful, these medicines could reduce the toxic RNAs that drive disease and improve muscle and neurological symptoms for people with myotonic dystrophy.
How similar studies have gone: Earlier small molecules like actinomycin D, microtubule inhibitors, and diamidines showed reversal of toxic RNA effects but were limited by toxicity, while preliminary data for two new MPCs show strong correction of splicing in DM1 and DM2 cell lines at nanomolar concentrations with limited toxicity.
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.