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PRMRP-Funded Myotonic Dystrophy Research

Since fiscal year 2018, myotonic dystrophy has been eligible for research funding through the U.S. Department of Defense Peer Reviewed Medical Research Program.

Federal investment advancing understanding and potential treatments for DM

MDF advocates and members of the DM community have helped make the case to Congress for continued PRMRP eligibility, giving researchers an opportunity to compete for significant federal research funding.

Through fiscal year 2024, PRMRP awards have directed more than $23 million to myotonic dystrophy research. These projects address a wide range of needs, from understanding the biological causes of DM1 and DM2 to investigating heart and brain involvement, biomarkers, experimental models, and possible therapeutic approaches.

PRMRP awards for myotonic dystrophy research

Partnering PI awards are presented together as one research project. Individual award amounts are provided beneath each investigator.

PRMRP grants are selected and administered by the federal government through a competitive review process. MDF does not select the investigators or administer these awards.

At a glance:

  • More than $23 million awarded
  • 13 funded research projects
  • 18 individual investigator awards
  • Funding awarded from FY2018 through FY2024

The Role of H19X Noncoding RNAs in the Pathogenesis of Myotonic Dystrophy Type I

Award mechanism: Discovery Award

Principal investigator: Yu Liu
Institution: University of Houston
Location: Texas
Award amount: $431,750

New Biological and Therapeutic Targets for Myotonic Dystrophy Type 1

Award mechanism: Investigator-Initiated Research Award, Partnering PI Option

  • J. Andrew Berglund, University at Albany, State University of New York, New York: $1,938,711
  • Kaalak Reddy, University at Albany, State University of New York, New York: $1,179,730

Combined project funding: $3,118,441

Foundational Studies to Understand Phenotypic, Region-Specific, and Cell-Specific Dysregulation in the DM Central Nervous System

Award mechanism: Investigator-Initiated Research Award, Partnering PI Option

  • Eric T. Wang, University of Florida, Florida: $1,525,000
  • John Day, Stanford University, California: $1,291,660

Combined project funding: $2,816,660

Noninvasive Identification of Cardiac Conduction System Abnormalities in a Myotonic Dystrophy Model

Award mechanism: Discovery Award

Principal investigator: Carly S. Filgueira
Institution: The Methodist Hospital Research Institute
Location: Texas
Award amount: $323,000

Development of Clinical Candidates for the Treatment of Myotonic Dystrophy

Award mechanism: Expansion Award, Funding Level 3

Principal investigator: Matthew Disney
Institution: University of Florida
Location: Florida
Award amount: $5,796,000

Understanding and Targeting Common Mechanisms of Myotonic Dystrophy Types 1 and 2 Using Novel Repeat Expansion Mouse Models

Award mechanism: Investigator-Initiated Research Award, Partnering PI Option

  • Maurice S. Swanson, University of Florida, Florida: $1,524,836
  • Laura P. Ranum, University of Florida, Florida: $1,525,000

Combined project funding: $3,049,836

Production of Adeno-Associated Virus in a Plant-Based System for the Treatment of Myotonic Dystrophy

Award mechanism: Discovery Award

Principal investigator: Stephen Streatfield
Institution: Fraunhofer USA Center for Molecular Biotechnology
Location: Delaware
Award amount: $260,698

Cardiac Manifestations in Myotonic Dystrophy Type 1: Arrhythmia Mechanisms and Novel Therapeutic Approaches

Award mechanism: Investigator-Initiated Research Award, Partnering PI Option

  • Mohamed Boutjdir, The Narrows Institute for Biomedical Research, New York: $1,189,999
  • Mohamed Chahine, Laval University, Québec, Canada: $1,099,960

Combined project funding: $2,289,959

Extracellular RNA Biomarkers of Myotonic Dystrophy

Award mechanism: Investigator-Initiated Research Award

Principal investigator: Thurman Wheeler
Institution: Massachusetts General Hospital
Location: Massachusetts
Award amount: $1,718,139

Extracellular Vesicles as Therapeutic Vehicles for Myotonic Dystrophy

Award mechanism: Discovery Award

Principal investigator: Thurman Wheeler
Institution: Massachusetts General Hospital
Location: Massachusetts
Award amount: $333,552

Role of Neuron-Specific Giant Ankyrin Isoforms in Developing Cardiac Arrhythmia in Myotonic Dystrophy Type 1

Award mechanism: Discovery Award

Principal investigator: Chaitali Misra
Institution: University of Illinois at Urbana-Champaign
Location: Illinois
Award amount: $317,200

Design and Study of Small Molecules That Cleave the RNA That Causes Myotonic Dystrophy Type 1

Award mechanism: Investigator-Initiated Research Award, Partnering PI Option

  • Matthew D. Disney, Scripps Research Institute, Florida: $2,081,250
  • Eric T. Wang, University of Florida, Florida: $571,875

Combined project funding: $2,653,125

The Role of Epigenetics and Methylation Near the DMPK Gene in Causing Neurodegeneration in Myotonic Dystrophy Type 1

Award mechanism: Discovery Award

Principal investigator: Steven Finkbeiner
Institution: J. David Gladstone Institutes
Location: California
Award amount: $377,600

Help keep DM federal research funding moving forward

Eligibility for PRMRP funding is determined through the annual congressional appropriations process. When people living with DM, family members, researchers, and other advocates speak to Congress, they help demonstrate the urgent need for continued federal investment in myotonic dystrophy research.