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UR Wellstone Research Projects

Dr. Thornton2013-2018 (Cycle 3)

The goals of this current Cycle are to:

  1. Investigate the causes of the multisystem manifestations of DM1 and to continue to develop new treatments
  2. Prepare for future therapeutic trials by identifying the best clinical endpoints and biomarkers to monitor treatment response.

To accomplish these goals, we have gathered support from key stakeholders (patients, family members, advocacy groups, pharmaceutical companies, Muscular Dystrophy Association, researchers, and NIH) to develop a Myotonic Dystrophy Clinical Research Network (DM CRN). Clinical studies are underway to help us prepare for new treatment trials. Studies take place at the 6 research centers participating in the DM CRN: University of Rochester, Kansas University Medical Center, Ohio State University, National Institutes of Health, Stanford University, University of Florida.

2008-2013 (Cycle 2)

Dr. Moxley and Christine Quinn

Through the projects in this Cycle, we gained new insights into how DM symptoms change over time and we identified promising experimental therapies that reverse myotonia and muscle fiber alterations in mouse models of myotonic dystrophy type 1 (DM1).

2003-2008 (Cycle 1)

The University of Rochester Wellstone MDCRC was designed to merge scientific and clinical research and learn more about myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD). Activities in this Cycle focused on how DM occurs and the identification of new treatments in DM.