"People have worried about side effects of daily prednisone, and many people have felt there must be a better alternative," said Griggs, who is professor of Neurology, Medicine, Pediatrics, Pathology and Laboratory Medicine, and a member of the Center for Human Experimental Therapeutics. "Our study is designed to address exactly this question: What is the best balance between effective treatment and side effects?"
Children will be evaluated every six months for three to five years. Researchers will measure the breathing capacity of the patients, which is often an indicator of how long a patient will live; they will ask patients and parents how satisfied they are with the treatment; and they will measure how long it takes patients to stand up after lying down, which is an indicator of how long a patient will be able to walk.
"With a healthy child, if you clap your hands and tell him to stand up as quickly as he can, he's up on his feet in less than a second. With a child with Duchenne muscular dystrophy, it might take 30 times longer," said Griggs. "The child will roll onto his stomach, laboriously lift his bottom into the air, and slowly move his arms to one knee, then the other, bracing himself until he finally straightens up."
The study brings together two worldwide groups of physicians who specialize in the study of muscle disorders. The Muscle Study Group, with nearly three dozen sites in the United States, Canada, and Europe, is based in Rochester and headed by Griggs. The TREAT-NMD study group is a consortium of dozens of institutions across Europe and elsewhere that work together to study neuromuscular disorders. TREA
|Contact: Tom Rickey|
University of Rochester Medical Center