New Muscular Dystrophy Cooperative Research Centers Announced
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), the National Institute of Neurological Disorders and Stroke (NINDS) and the National Institute of Child Health and Human Development (NICHD), a part of the National Institutes of Health, have funded three new Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers focusing on this group of genetic muscle-wasting diseases. The late Senator Wellstone was a major champion of muscular dystrophy issues in the Congress.
The center at the University of Pennsylvania, co-directed by the university’s H. Lee Sweeney, Ph.D., and Kathryn R. Wagner, M.D., Ph.D., of The Johns Hopkins School of Medicine, will explore new strategies for treating a variety of muscular dystrophies (MD). Two laboratory projects are focused on ways to increase muscle growth, and another on examining compounds that may be able to inhibit enzymes involved in breaking down muscle tissue. Clinical trials will determine the safety and feasibility of a potential drug treatment for Duchenne Muscular Dystrophy (DMD). The core facility — a muscle physiology lab — will analyze MD mouse models. Other sites involved with this center are the University of Florida, Gainesville, and the NINDS Intramural Research Program.
Center scientists at Washington, D.C.’s Children’s National Medical Center, under the direction of Eric P. Hoffman, Ph.D., and Diana M. Escolar, M.D., will study biochemical pathways that contribute to DMD. A clinical project aims to identify genetic modifiers of the disease, and two laboratory studies focus on muscle cell damage and muscle growth and remodeling in mice. The center also has a bioinformatics and computing core, as well as a clinical core to help support the Cooperative International Neuromuscular Research Group, an existing consortium of MD clinical investigators. Collaborating with the center is the University of Padova in Italy.
The center at the University of Iowa, directed by the university’s Kevin P. Campbell, Ph.D., and Steven A. Moore, M.D., Ph.D., will explore therapeutic strategies for different muscular dystrophies. One project focuses on muscle membrane maintenance and repair, and another on potential embryonic stem cell treatment in mice. A third study involves people with Fukutin Related Protein MD and the development of a mouse model for further investigation of this disease. The center cores here will serve as a national resource for human muscle biopsies, fibroblast cell cultures and embryonic stem cells for MD researchers, and will provide advanced services for diagnosing the different dystrophies.
These new centers join three others already funded by the National Institutes of Health and the Muscular Dystrophy Association at the University of Washington, the University of Pittsburgh and the University of Rochester. The centers spring from the Muscular Dystrophy Community Assistance, Research and Education (MD-CARE) Act passed by Congress in 2001. The centers work individually and collaboratively, and are guided by a steering committee that includes representatives from each center. Each has both basic and clinical research projects, and one or more core facilities to support them. Centers must also make core resources or services available to the national muscular dystrophy research community.
MD is characterized by progressive weakness and degeneration of the skeletal or voluntary muscles which control movement. Researchers at the Senator Paul D. Wellstone Centers study various forms of MD, including Duchenne/Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Dystrophy, Limb-Girdle Muscular Dystrophy, and others. MD can affect people of all ages. Although some forms first become apparent in infancy or childhood, others may not appear until middle age or later.
NIAMS, NINDS and NICHD are part of the U.S. Department of Health and Human Services’ National Institutes of Health (NIH). The mission of the NIAMS is to support research into the causes, treatment and prevention of arthritis and musculoskeletal and skin diseases; the training of basic and clinical scientists to carry out this research; and the dissemination of information on research progress in these diseases. For more information about NIAMS, call the information clearinghouse at (301) 495-4484 or (877) 22-NIAMS (free call) or visit the NIAMS Web site at www.niams.nih.gov. The mission of the NINDS is to reduce the burden of neurological disease — a burden borne by every age group, by every segment of society, by people all over the world. For more information about NINDS, contact the Information Center at (800) 352-9424 or visit the NINDS Web site at www.ninds.nih.gov. The mission of the NICHD is to ensure that every person is born healthy and wanted, that women suffer no harmful effects from reproductive processes, and that all children have the chance to achieve their full potential for healthy and productive lives, free from disease or disability, and to ensure the health, productivity, independence, and well-being of all people through optimal rehabilitation. For more information about NICHD, call the Information Resource Center at (800) 370-2943 or visit the NICHD Web site at www.nichd.nih.gov.
The National Institutes of Health (NIH) — The Nation’s Medical Research Agency — includes 27 Institutes and Centers and is a component of the U. S. Department of Health and Human Services. It is the primary Federal agency for conducting and supporting basic, clinical, and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.
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