Posted February 26, 2013
Avital Cnaan, Ph.D., Children's Research Institute at Children's National Medical Center
Although there is no cure for DMD, several new therapies are under development. Current clinical trial outcome measurements, such as muscle strength, are not, however, correlated well with patient function or quality of life. Dr. Avital Cnaan and her research team at Children's National Medical Center will build upon the existing DMD natural history study to identify clinical trial outcome measurements that can be utilized in the planning of DMD clinical trials. The long-term goal of Dr. Cnaan's research is the development of practical and easily administered outcome measures that are sensitive and responsive to changes produced by treatments in children and adults with muscular dystrophies or other neuromuscular diseases across the lifespan and stages of disease severity.