Progression of Friedreich ataxia: quantitative characterization over 5 years
Annals of Clinical and Translational Neurology2016Vol. 3(9), pp. 684–694
Citations Over TimeTop 10% of 2016 papers
Maya Patel, Charles J. Isaacs, Lauren Seyer, Karlla W. Brigatti, Sarah Gelbard, Cassandra J. Strawser, Debbie Foerster, Julianna Shinnick, Kimberly Schadt, Eppie M. Yiu, Martin B. Delatycki, Susan Perlman, George Wilmot, Theresa A. Zesiewicz, Katherine D. Mathews, Christopher M. Gómez, Grace Yoon, S. H. Subramony, Alicia Brocht, Jennifer Farmer, David R. Lynch
Abstract
While age predicted progression speed in simple analyses and may provide an effective way to stratify cohorts, separating the effects of age and genetic severity is difficult. Controlling for baseline severity, GAA is the major determinant of progression rate in FRDA. Clinical trials will benefit from enrollment of younger subjects, and sample size requirements will shrink with longer assessment periods. These findings should prove useful in devising gene therapy trials in the near future.
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