The Myotonic Dystrophy Foundation made the following grants in 2026:
2026 MDF Early Career Scholars:
Samuel Carrell, MD, PhD
Assistant Professor of Neurology
Virginia Commonwealth University, United States
Individuals with DM1 show variable symptoms affecting muscles, heart, and cognition, with age of onset and severity poorly predicted by repeat length alone. This variability complicates clinical care and trial design. The project “Identifying Genetic Modifiers of Disease Severity in Myotonic Dystrophy” aims to identify genetic modifiers that influence disease manifestations by systematically knocking out genes in patient-derived muscle progenitor cells and measuring changes in disease activity. Genes identified will be validated individually in DM1 and control cells. These studies will reveal factors that exacerbate or protect against DM1 symptoms, providing targets for future research and potential therapeutic development.
Katarzyna Taylor, PhD
Assistant Professor
Adam Mickiewicz University, Poznan, Poland
DM1 is a genetic disorder affecting multiple organs with no current treatment to slow progression. Many symptoms arise from reduced levels of MBNL1 and MBNL2 proteins, which are essential for normal cellular function. Restoring MBNL levels offers therapeutic potential, but precise control is critical to avoid harmful effects. This research project “Mechanisms of MBNL Regulation: Molecular Targets for Potential Therapeutic Intervention in DM1” aims to identify regulatory mechanisms controlling MBNL1 and MBNL2 expression, providing new targets for therapy. Understanding these mechanisms could enable fine-tuned treatments, alone or in combination, and accelerate development of personalized interventions tailored to disease stage, progression, and individual patient needs.
2026 MDF High Priority:
Curtis Nutter, PhD
Assistant Professor
University of Missouri, Columbia, MO, United States
DM1 affects multiple organs, with brain symptoms such as fatigue, cognitive difficulties, and low motivation severely impacting daily life. Current methods cannot reliably measure these symptoms or treatment effects. This project focuses on the choroid plexus, which produces cerebrospinal fluid (CSF), and its abnormal RNA splicing in DM1. The study “Choroid plexus RNA Mis-splicing Drives Cerebrospinal Fluid Biomarker Changes in DM1” will analyze brain and CSF samples from patients and use lab-grown brain tissues to test treatments. The goal is to identify CSF biomarkers that reflect brain dysfunction, enabling better disease monitoring, evaluation of therapies, and development of new brain-targeted treatments to improve quality of life for people with DM1.