The Myotonic Dystrophy Foundation UK began grantmaking activities in 2015. The Myotonic Dystrophy Foundation UK has formed an alliance with the Myotonic Dystrophy Foundation in the United States to support selected basic and translational research projects, as a component of its efforts to accelerate therapy development. Information on the Myotonic Dystrophy Foundation is available here. Information on the Myotonic Dystrophy Foundation is available here.
Research Grant Awards
In partnership with the Myotonic Dystrophy Foundation in the United States, the Myotonic Dystrophy Foundation UK supports scientific investigations to enhance the quality of life of people living with myotonic dystrophy (DM) and advance research focused on finding treatments and a cure for this disease. As a part of this effort, the partner organizations offer several funding opportunities for DM researchers and support research efforts.
2020 Grant Recipients
- “Reach DM- Study to Promote Trial Readiness by Genetic Analysis and Telemedicine Assessments”
PI: Johanna Hamel, MD, University of Rochester, New York, US
2018 Grant Recipients
- “Request for Support for Publication and Open Access Fee for a Peer-Reviewed Myotonic Dystrophy Therapy Review Paper”
PI: Ruben Aretero, PhD, University of Valencia, Spain
- “Myotonic Dystrophy Clinical Research Network (DMCRN) Site Grants: Multicentre Study of Natural History and Genetic Modifiers in Myotonic Dystrophy Type 1”
PI: Nicholas Johnson, MD, Virginia Commonwealth University, Virginia, US
2017 Grant Recipients
- “Biomarker Qualification Project”
Jane Larkindale, DPhil, Critical Path Institute, US
2016 Grant Recipients
- “Workshop Support - Myotonic Dystrophy: Developing a European Consortium for Care and Therapy”
PI: Alexandra Breukel, PhD, European Neuromuscular Centre, Netherlands
- “Prevalence of Myotonic Dystrophy”
PI: Nicholas E. Johnson, MD, University of Utah, US
- “PHENO-DM1- Myotonic Dystrophy type 1 (DM1) Deep Phenotyping to Improve Delivery of Personalized Medicine and Assist in the Planning, Design and Recruitment of Clinical Trials”
PI: Hanns Lochmüller, MD, Newcastle University, UK
- “Development of Magnetic Resonance Imaging as an Endpoint in Myotonic Dystrophy Type 1”
PI: Donovan Lott, PhD, University of Florida, US
- “Building a Better Mouse”
PI: Cathleen Lutz, PhD, The Jackson Laboratory, US
- “DM Cell Line Library”
PI: Michael Sheldon, PhD, RUCDR Infinite Biologics, Rutgers University, US
- “Extracellular RNA as Biomarkers of Myotonic Dystrophy”
PI: Thurman Wheeler, MD, Massachusetts General Hospital, US
2015 Grant Recipients
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“Inhibiting Transcription of CUG/CCUG Expanded Repeats with Small Molecules”
PI: Andy Berglund, PhD (University of Florida), PI: Paul August, PhD (Sanofi)
Myotonic Dystrophy Foundation UK Research Fellows
In 2015, the Myotonic Dystrophy Foundation UK began providing support to the Myotonic Dystrophy Foundation in United States’ very successful Research Fellowship programme. This programme provides two-year pre- and postdoctoral research fellowships to support new and innovative studies relevant to the pathogenesis of DM, disease progression, best practices in clinical management of the disorder, and therapeutic and diagnostic development for myotonic dystrophy. Through this programme, the partner organizations support up-and-coming pre- and postdoctoral fellows around the world to expand the base of committed DM researchers. The goal of the Research Fellows programme is to support adequate early career and ongoing funding in order to establish and drive a robust professional research community with a long-term commitment to the disease. The specific objectives that the Research Fellows programme focuses on to achieve those goals are to:
- Support young investigators pursuing DM discovery
- Increase the scope and quality of publications of DM research
Since the programme began, there has been 34 funded fellows from 17 different distinguished institutions in five countries. Many former fellows have remained in the DM research field after completing their fellowships and cited that they chose to continue in the field due to their interaction with the community, interest in the disease, and the research skills they gained. The Myotonic Dystrophy Foundation UK has supported the following fellows:
2021 Fellows
- “Mechanisms of DM1 Cardiac Pathogenesis and Potential Therapeutics
Rong-Chi Hu, Baylor College of Medicine, Houston, Texas, US
- “Brain Choroid Plexus Dysregulation and Cerebral Atrophy in DM1”
Benjamin M. Kidd, University of Florida, Gainesville, Florida, US
- “Discovery of Dietary Natural Compounds as Potential Therapeutics for Myotonic Dystrophy (DM)”
Subodh Kumar Mishra, PhD, The RNA Institute, University of Albany, New York, US
2020 Fellows
- “Targeting the RNA that Causes DM2 for Degradation with Small Molecules”
Raphael Benhamou, PhD, Scripps Research Institute, Florida, US
- “Novel Strategy Targeting Muscle Stem Cells as a Therapeutic Approach for Myotonic Dystrophy Type 1”
Talita Conte, PhD, University of Montreal, Canada
- “Improving the Activity of Diamidines for Potential Therapeutic Use for Patients with Myotonic Dystrophy Types 1 and 2”
Jana Jenquin, PhD, University of Florida, Gainesville, Florida, US
- “Restoration of MBNL Proteins Through miRNA Blocking as DM1 Therapy”
Sarah Overby, Incliva, University of Valencia, Spain
2019 Fellows
- “Selective and Non-toxic Small Molecules that Cleave r(CUG) Repeats in DM1—Optimization and Evaluation as a Therapeutic Approach”
Shruti Choudhary, PhD, Scripps Research Institute Florida, US
- “Engineering Synthetic RNA Binding Proteins to Probe the Mechanisms of Myotonic Dystrophy and Development of Potential New Therapeutics”
Carl Shotwell, University of Florida, Gainesville, Florida, US
2018 Fellows
- “CRISPRI-induced Transcriptional Silencing of DMPK as a Therapeutic Strategy Against Myotonic Dystrophy Type 1”
Florent Porquet, University of Liege, Belgium
- “Congenital Myotonic Dystrophy: Pathomechanism and Therapeutic Development”
Curtis Nutter, PhD, University of Florida, Gainesville, Florida, US
- “Tissue Specific Expression of Expanded CUG Repeat RNA to Investigate the Cardiac Pathogenesis of Myotonic Dystrophy Type 1”
Ashish Rao, Baylor College of Medicine, Houston, Texas, US
- “Molecular Characterization of RNA and RAN Protein Effects in DM2”
Kiruphagaran Thangaraju, PhD, University of Florida, Gainesville, Florida, US
2017 Fellows
- “Mechanism of CNS-associated Behavioral Dysfunction in Novel Mouse Model of Myotonic Dystrophy Type 1”
Anwesha Banerjee, PhD, Emory University, Atlanta, Georgia, US
- “Pre-clinical Investigations of Small Molecule-mediated Targeting of Toxic RNA Production in DM2”
Kaalak Reddy, PhD, University of Florida, Gainesville, Florida, US
2016 Fellows
- "Structural and Functional Connectivity in the Brains of Patients with Adult and Late Onset Myotonic Dystrophy Type 1 (DM1): A Potential Biomarker for Disease Progression"
Ian DeVolder, PhD, University of Iowa, US
- “Evaluation of Functional Connectivity as a Brain Biomarker in Congenital Myotonic Dystrophy”
Melissa M. Dixon, PhD, University of Utah, US
- “A 14‐year Longitudinal Study of Cognition and Central Nervous System Involvement in Adult and Late‐onset Phenotypes of Myotonic Dystrophy Type 1”
Benjamin Gallais, PhD, Universite de Sherbrooke, Canada
- “Mechanisms of Skeletal Muscle Wasting Caused by Expanded CUG Repeat RNA”
Ginny R. Morriss, PhD, Baylor College of Medicine, Houston, Texas, US
- “A New Approach of Pathomolecular Mechanism in Myotonic Dystrophy Insulin Resistance by Nutrigenomics”
Laura Valentina Renna, PhD, IRCCS-Policlinico San Donato, Italy
- “Myotonic Dystrophy Type 2: Mouse Models, Pathomechanism and Therapy”
Lukasz Sznajder, PhD, University of Florida, Gainesville, Florida, US
2015 Fellows
- “Studying Genome-Wide MBNL-RNA Structure Interactions in Neuronal Development and DM”
Ranjan Batra, PhD, University of California, San Diego, California, US
- “Precise Lead Therapeutics for Myotonic Dystrophy via in cellulo Synthesis”
Viachaslau Bernat, PhD, Scripps Research Institute Florida, US
- “An Investigation of the Cellular and Microbial Etiologies of Gastrointestinal Pathologies in Myotonic Dystrophy Zebrafish”
Melissa Hinman, PhD, University of Oregon, Eugene, Oregon, US
More Information
Additional award information will be posted to this page as it becomes available. For questions, please contact info@myotonic.org.