Funding sources for the network and the studies running on the network include: the FSHD Society, Muscular Dystrophy Association, private philanthropy, National Institute of Neurological Disorders and Stroke, Friends of FSHD Research, Institute of Myology Association, the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Fulcrum Therapeutics, and Dyne Therapeutics.
The FSHD Clinical Trial Research Network (CTRN), launched in 2016 with funding by the FSHD Society, is a cornerstone of our strategy to accelerate the development of treatments. Comprised of leading international FSHD research centers, the CTRN functions as a “lab without walls” with uniform standards and training for assessing patients and sharing data. A vitally important part of the infrastructure, the CTRN:
- Enables fundamental research to be done at scale and faster;
- Attracts biopharmaceutical companies into the FSHD field;
- Improves patient care by enforcing best practices and standards.
The ReSOLVE study, the largest study ever conducted in FSHD, is currently being run on the network, and all of the network sites are also involved in clinical trials of new therapies. A second study, MOVE, is running on the network to understand which measures taken by a doctor during a clinical visit translate into meaningful changes in patients' daily lives.
The FSHD Society will continue to invest in the CTRN to expand the network as needed to additional sites both within the U.S. and in Europe.
Current CTRN sites and principal investigators:
Sites in the United States
- Kennedy Krieger Institute, Baltimore MD — Doris Leung, MD PhD
- Stanford University School of Medicine — John W. Day, MD, PhD
- The Ohio State University Medical Center, Columbus, OH — Bakri Elsheikh, MD
- University of California Los Angeles — Perry Shieh, MD PhD
- University of Colorado — Matthew P. Wicklund, MD
- University of Florida Gainesville — S. H. Subramony, MD
- University of Kansas Medical Center, Kansas City KS — Jeffrey Statland, MD
- University of Rochester Medical Center, Rochester NY — Rabi Tawil, MD
- University of Texas Southwestern Medical Center — Jaya Trivedi, M.D.
- University of Utah, Salt Lake City, UT — Russell Butterfield, MD
- University of Washington, Seattle — Leo H. Wang, MD PhD
- Virginia Commonwealth University, Richmond, VA — Nicholas Johnson, MD
Collaborating European sites
- Radboud University, Nijmegen, The Netherlands – Karlien Mul, MD PhD, and Baziel van Engelen, MD PhD
- University of Milan, Italy – Valeria Sansone, MD
- University of Nice, France – Sabrina Sacconi, MD PhD