MDF’s pilot grants provide support for innovative, exploratory projects and preliminary studies to further myotonic dystrophy research. Congratulations to the 2026 Pilot Grant recipients: Dr. Jeffrey Statland, Dr. Ariadna Bargiela, and Dr. Karlien Mul!
Jeffrey Statland, M.D.
University of Kansas Medical Center, KS, United States
The project “Modeling Cardiomyopathy in DM1: A Pilot for Scalable Cardiovascular Tracking” aims to improve understanding of how heart disease develops and progresses in people with DM1 by creating a shared data resource that combines electronic health record information across multiple sites. Using this data, the researchers will study how patients respond to commonly used heart treatments over time and identify factors that may predict worse outcomes or improve survival.
Ariadna Bargiela, Ph.D
INCLIVA Health Research Institute, Valencia, Spain
The project “DM2 splice index pilot study and assessment of MBNL1-modulating therapeutics” aims to help prepare DM2 for future clinical trials by developing a new molecular test, the DM2 Splice Index, that can measure disease-related genetic changes in muscle cells and track whether treatments are working. The researchers will also test whether drugs already being developed for DM1, including ATX-01, currently in clinical trials for DM1, can improve these molecular abnormalities in DM2 patient-derived muscle cells.
Karlien Mul, M.D., Ph.D.
Radboud University Medical Center (Radboudumc), Nijmegen, Netherlands
The project “DM2-Activ: A Rasch-Built Patient-Reported Outcome Measure for Myotonic Dystrophy Type 2” will develop DM2-Activ, a new questionnaire designed specifically to measure how DM2 affects daily functioning. Researchers across 14 centers in nine countries will collect patient-reported data online and use Rasch analysis to create a reliable, sensitive tool for use in future natural history studies and clinical trials.
