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 *1}Department 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