Duchenne Muscular Dystrophy is one of the most common and devastating genetic diseases of childhood, affecting approximately 1 in 5000 boys. Progressive muscle degeneration leads to loss of ambulation by age 12, loss of upper arm use in the teen years, and heart and respiratory failure leading to death in the 20's. The Center for Duchenne Muscular Dystrophy at UCLA leads the nation in translational science focused on Duchenne and is the first comprehensive Duchenne clinic in the western United states. A leading group of UCLA scientists, clinicians and researchers is poised to achieve significant advances in research, clinical care and treatments for Duchenne that are improving, and will potentially extend, the lives of those afflicted.
CDMD co-directors Dr. Miceli and Dr. Nelson join Dr. McDonald in drafting a letter in support of accelerated approval for Ataluren, signed by 40 scientists, and sent to FDA leadership Friday. PDUFA Date October 24. #Bereasonable