Sunday, January 10, 2010

Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design

Movement Disorders, Early View (Articles online in advance of print)
Published Online: 8,Jan,2010

 Lisa S. Friedman, BS 1 2 3, Jennifer M. Farmer, MS 1 2 3, Susan Perlman, MD 4, George Wilmot, MD, PhD 5, Christopher M. Gomez, MD PhD 6 7, Khalaf O. Bushara, MD 6, Katherine D. Mathews, MD 8, S. H. Subramony, MD 9 10, Tetsuo Ashizawa, MD 9 11, Laura J. Balcer, MD, MSCE 1, Robert B. Wilson, MD, PhD 12, David R. Lynch 1 2 3 *1Department of Neurology, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania
2Department of Pediatrics, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania
3Children's Hospital of Philadelphia, Philadelphia, Pennsylvania
4Department of Neurology, David Geffen School of Medicine at the University of California, Los Angeles, Los Angeles, California, USA
5Department of Neurology, Emory University, Atlanta, Georgia, USA
6Department of Neurology, University of Minnesota, Minneapolis, Minnesota, USA
7Department of Neurology, University of Chicago, Chicago, Illinois, USA
8Departments of Neurology and Pediatrics, University of Iowa, Iowa City, Iowa, USA
9Department of Neurology, University of Texas Medical Branch, Galveston, Texas, USA
10Department of Neurology, University of Mississippi, Jackson, Mississippi, USA
11Department of Neurology, University of Florida, Gainesville, Florida, USA
12Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania

Keywords: Friedreich ataxia, natural history study, clinical neurology examination, mitochondrial disorder, trinucleotide repeat disease