In December 2024, after nine impactful years, the Trustees of Myotonic Dystrophy Foundation UK (MDF UK) made the difficult decision to cease operations, believing it was in the best interests of the charity and its mission.
MDF UK, originally established in 2015 as the Wyck Foundation, was founded in London by a dedicated group of individuals committed to supporting global efforts to improve the quality of life for those living with myotonic dystrophy (DM). From its inception, MDF UK focused on accelerating the discovery of treatments and driving advancements in DM care and research.
Over nearly a decade, MDF UK contributed significantly to the progress in the field of DM. The organisation funded 16 research grants and 22 fellowships, each playing a critical role in advancing understanding of this complex disease and bringing researchers closer to a cure.
MDF UK's sister organisation, the Myotonic Dystrophy Foundation (MDF) in the United States, remains steadfast in carrying forward the mission that MDF UK so passionately championed. The Trustees are confident that MDF will continue to address the needs originally targeted by MDF UK and will expand upon this important work. With a global perspective, MDF is dedicated to uniting the DM community, driving research, and investing in solutions that benefit individuals worldwide.
Thank you to all the donors who supported MDF UK over the years. Their generosity and commitment made it possible to fund groundbreaking research and support critical advancements. Special recognition goes to those who donated in memory or honor of loved ones—these contributions have left a lasting legacy, propelling the field closer to better treatments and a cure for myotonic dystrophy.
As MDF UK concludes its journey, the community is encouraged to continue supporting MDF's global efforts to improve care and find a cure. Honoring the legacy of MDF UK ensures that meaningful progress continues for everyone affected by DM. Click here to support MDF! >>>
Funding the Future of Myotonic Dystrophy Research
From its inception in 2015, the Myotonic Dystrophy Foundation UK was instrumental in advancing research efforts aimed at improving the lives of individuals with myotonic dystrophy. The organisation focused on:
- Supporting basic and translational research to discover treatments and a cure for myotonic dystrophy.
- Advocating for better access to care and enhancing the regulatory pathway for therapeutic advancements.
In collaboration with the Myotonic Dystrophy Foundation in the United States, MDF UK provided critical funding to a variety of research projects, aiming to accelerate therapy development. This partnership included support for the highly successful Research Fellowship Programme, which offers two-year pre- and postdoctoral research fellowships. These fellowships focus on innovative studies related to disease pathogenesis, progression, clinical management, and therapeutic development for DM.
Many of these fellows have continued their commitment to DM research, citing the invaluable skills and community connections they gained through the programme. The fellows supported by MDF UK have made significant contributions to advancing the understanding and treatment of DM.
Learn more about the awardees who have benefited from MDF UK’s support, showcasing their achievements and ongoing impact in the field.
2021 Research Fellowships
- “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 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
2020 Research Fellowships
- “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
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
2019 Research Fellowships
- “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 Research Fellowships
- “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 Grant Recipients
- “Biomarker Qualification Project”
Jane Larkindale, DPhil, Critical Path Institute, US
2017 Research Fellowships
-
“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 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
2016 Research Fellowships
- "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 Grant Recipients
- “Inhibiting Transcription of CUG/CCUG Expanded Repeats with Small Molecules”
PI: Andy Berglund, PhD (University of Florida), PI: Paul August, PhD (Sanofi)
2015 Research Fellowships
- “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
Current Funding Opportunities
Myotonic dystrophy focused research professionals are encouraged to explore MDF's current Grant and Research Fellowship opportunities. Please direct all research funding based questions to grants@myotonic.org.