Quantcast

University of Massachusetts Medical School Opens New Center for FSHD Research and Training

November 15, 2013

FSH Society partners with Wellstone Muscular Dystrophy Cooperative Research Center.

Boston, Massachusetts (PRWEB) November 15, 2013

A new cooperative research center focusing on Facioscapulohumeral Muscular Dystrophy (FSHD) has been established at the University of Massachusetts Medical School (UMMS). The National Institutes of Health Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center will focus its research and training programs on FSHD, one of the most prevalent myopathies affecting both children and adults.

The Wellstone Center at UMMS will collaborate with the FSH Society, a world leader in combating FSHD that helps connect patients and families with clinical centers across the country. FSH Society President and CEO Daniel Paul Perez was a leading figure in the early advocacy efforts around FSHD and co-authored the MD CARE Act bill and helped compete for the initial $9 million in NIH funding for the UMMS Wellstone Center. Perez, who himself has FSHD, has been a national leader for patients with FSHD over the last thirty years.

There are currently no clinical trials of novel therapeutics for FSHD ongoing in the United States because of a lack of relevant preclinical data to direct development of therapeutic targets to disease mechanisms. The Wellstone Center seeks to change that.

“FSHD leads to severe muscle weakness and our Center's therapeutic approaches will focus on development of drugs that target disease genes, as well as drugs that improve muscle strength,” says Center Director Charles P. Emerson Jr, PhD, professor of cell & developmental biology and neurology. “The therapeutic approaches being developed by our center will have broad application to the treatment of other muscular dystrophies and other debilitating medical conditions of muscle weakness including aging, muscle injury and confined bed rest.”

“The new partnership between the Wellstone Center and other translational research groups at UMMS, such as the Department of Neurology and the Gene Therapy Center, have given the programs another opportunity to bring their unique talents to bear on a vexing clinical problem,” said Terence R. Flotte, MD, executive deputy chancellor, provost and dean of the school of medicine. “This is an important addition to the translational research enterprise.”

The UMMS Center’s mission includes training the next generation of muscular dystrophy clinical and basic researchers, and providing the broader research community with biomaterials for muscular dystrophy research. An important new initiative for the Center will be the establishment of an FSHD clinic, which will serve patients in the region and enable them to participate in research and clinical trials. The partnership between FSHD patients, their families, Center scientists, clinicians and the overall industry will be essential for the Center to successfully carry out its mission to develop therapies for FSHD and other debilitating muscle diseases.

Additionally, the Center supports an Education and Training Core that will oversee a pre- and postdoctoral fellowship program designed to mentor young scientists for careers in skeletal muscle and muscular dystrophy research, with a strong focus on multidisciplinary training for FSHD and muscle disease research. A Cell Core laboratory, with a repository of muscle tissue and cells, will perform genomic studies to establish FSHD biomarkers and will make cell lines available to investigators worldwide for testing new FSHD therapeutics.

The FSH Society will work directly with Wellstone Center leadership to consult on key communications, organize patient and research meetings and workshops, and provide education and support for patients and families participating in the Center’s studies.

The UMMS Wellstone Center is one of six centers funded by the National Institutes of Health, with additional funding from foundations and individuals. These centers engage in translational research with patients and industry to develop novel therapies for muscular dystrophies – genetic diseases resulting in the degeneration of muscle or muscle function. A 2001 federal law, called the MD CARE Act, directed the NIH to establish centers of excellence for research on muscular dystrophy; the program was subsequently named in honor of the late Senator Paul D. Wellstone of Minnesota, a champion of the NIH and muscular dystrophy research.

# # #

About the FSH Society

The FSH Society, founded in 1991 by two FSHD patients, is a world leader in combating muscular dystrophy. The non-profit has provided millions of dollars in seed grants to pioneering research worldwide, creating an international collaborative network of patients and researchers. The FSH Society seeks to serve as a source of information and support for all patients and families with FSHD; act as a driving force in the development of research directed towards treatments and ultimately a cure; and bring support to patients and research for FSHD through effective engagement of governmental and private sector organizations and entities. For five consecutive years, the Society has received the Charity Navigator’s four-star rating, the highest distinction held by less than four percent of non-profit organizations in the country. The FSH Society offers a community of support, news and information for FSHD patients and families through its website at http://www.fshsociety.org. For more information about FSHD, please contact the Society at 781-301-6649.

For the original version on PRWeb visit: http://www.prweb.com/releases/2013/11/prweb11338086.htm


Source: prweb



comments powered by Disqus