New small molecule therapeutics to help 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-11617693 · Data updated 2026-11-12
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.
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) | $13,263 |
| NIH project number | 3R01NS120485-05S1 |
| Project ID | NIH-11617693 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are patients with myotonic dystrophy or related repeat expansion disorders whose disease mechanism involves toxic RNA activity that could be targeted by new drugs.
✕Less likely to help
Patients without a disease driven by RNA repeat expansion or those not eligible for future trials may not benefit.
What this research studies
This project focuses on creating and testing modified polycyclic compounds that may decrease the RNA troublemakers responsible for myotonic dystrophy. Researchers will modify chemical structures to balance efficacy and safety, guided by prior hints that some compounds can lower toxic RNAs in laboratory models. The work includes evaluating how these drugs affect RNA processing pathways and disease-related cellular problems in DM cell lines. If successful, these compounds could become the next step toward therapies for people with DM and similar disorders.
Why it matters
Potential benefit: If successful, the research could lead to new drug candidates that reduce harmful RNA activity and improve function for people with myotonic dystrophy.
How similar studies have gone: Previous small molecules showed promising RNA reduction and splicing rescue in lab models, but many had toxicity; the new MPCs aim to overcome these issues.
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.
-
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.
-
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.
-
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.