"Based on the current pace of therapeutic development, it is reasonable to expect that new therapies will be advanced into clinical trials in the near future, and the new network will ensure that we're prepared for that."
TUCSON, Ariz. (PRWEB) January 23, 2013
The Muscular Dystrophy Association (MDA) has launched a clinical research network that will provide an infrastructure for trials of new treatments for myotonic muscular dystrophy (MMD, also known as DM). The rapid pace of research in this disease was the impetus behind the development of the network.
Each center in the new network has a particular interest and expertise in MMD, and the centers are distributed across the United States to maximize opportunities for participation in studies. Neurologist Charles Thornton at the University of Rochester Medical Center is the overall principal investigator for the network.
There are five university-based centers with expertise in this disorder that make up the network. Their first task will be to determine the usual progression of myotonic dystrophy and the best ways to measure the response of the disease to experimental treatments now in the developmental pipeline.
The five centers are:
Stanford University School of Medicine, Stanford, Calif. | Director: John Day, M.D., Ph.D.
University of Florida College of Medicine, Gainesville, Fla. | Director: Tetsuo Ashizawa, M.D.
University of Kansas Medical Center Research Institute, Kansas City, Kan. | Director: Richard Barohn, M.D.
University of Rochester Medical Center, Rochester, N.Y. | Director: Charles Thornton, M.D.
Ohio State University Medical Center, Columbus, Ohio | Director: John Kissel, M.D.
Network a response to scientific progress
"Recent years have seen rapid progress in the development of molecularly targeted therapy for myotonic dystrophy," said Jane Larkindale, MDA's director of translational research. "Basic research has shown that this disorder involves a disease mechanism known as 'RNA toxicity,' and translational research has shown that this mechanism is unusually responsive to therapeutic intervention. Based on the current pace of therapeutic development, it is reasonable to expect that new therapies will be advanced into clinical trials in the near future, and the new network will ensure that we're prepared for that."
About myotonic muscular dystrophy
Myotonic muscular dystrophy (MMD) is a common form of muscular dystrophy that weakens skeletal muscles and also can affect many other organs in the body, including the heart, eyes, brain and gastrointestinal tract. Caused by an abnormal expansion of DNA on either chromosome 19 or chromosome 3, the disease also is known as "dystrophia myotonica," or "DM."
For more on MMD and current research, see:
- MMD Research: Seeking to Free Proteins from a 'Toxic Web', MDA’s Quest magazine, July 2012
- In Focus: Myotonic Muscular Dystrophy, MDA’s Quest magazine, April 2012
MDA is the nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education.
In addition to funding some 300 research projects worldwide, MDA maintains a national network of 200 medical clinics; facilitates hundreds of support groups for families affected by neuromuscular diseases; and provides local summer camp opportunities for thousands of youngsters living with progressive muscle diseases.
For more information, visit mda.org and follow MDA on Facebook (facebook.com/MDAnational) and Twitter (@MDAnews).