A large international study aimed at improving the care of muscular dystrophy patients worldwide is being launched by physicians, physical therapists, and researchers at the University of Rochester Medical Center.
Neurologist Robert "Berch" Griggs, M.D., is heading the study of treatments for Duchenne muscular dystrophy, the most common form of the disease that affects children. The condition, which affects boys almost exclusively, progresses rapidly. Boys' symptoms start when they are toddlers; untreated, they end up in a wheelchair before they become teenagers. With today's best treatments, the disease, which affects about 28,000 boys and young men in the United States, is often fatal by the time a patient reaches his 20s or early 30s.
Despite decades of research, Griggs calls the current treatment landscape for the disease "chaotic." Recently he has identified 29 different treatment regimens in use by doctors around the world.
The new study is designed to eliminate the chaos. With at least $11 million in funding from the National Institute of Neurological Disorders and Stroke, Griggs and co-leader Kate Bushby, M.D., and investigators at 41 other institutions around the world will study the three treatments most commonly used today. The study will include 300 boys ages 4 through 7 throughout North America and Europe. Recruiting for the study will begin in the summer of 2011. Study funding is slated to reach close to $15 million.
The central feature of Duchenne muscular dystrophy is muscle weakness. The first symptoms, often seen around age 2 or 3, usually come when a boy has difficulty running, jumping, or climbing stairs. From there, even with treatment, the disease progresses very quickly. Boys are often confined to a wheelchair by age 9 or 10 because of weakness in their legs; their breathing muscles begin to fail and their heart muscle weakens dramatically in their teen years. Most patients begin receiving assisted brea
|Contact: Tom Rickey|
University of Rochester Medical Center