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- Title
Measuring Disease Progression in Giant Axonal Neuropathy: Implications for Clinical Trial Design.
- Authors
Roth, Lisa A.; Marra, Jonathan D.; LaMarca, Nicole H.; Sproule, Douglas M.
- Abstract
As part of a natural history study of giant axonal neuropathy, we hypothesized that the Friedreich Ataxia Rating Scale and the Gross Motor Function Measure would show a significant change over 6 months, reflecting subjects’ decline in motor function. The Friedreich Ataxia Rating Scale was performed on 11 subjects and the Gross Motor Function Measure was performed on 10 subjects twice with a six-month interval. A paired two-tailed t-test was used to assess the difference in each subject’s score. Significant changes were found over six months of 11.7 ± 11.0 (P = 0.006) for the Friedreich Ataxia Rating Scale and –10.0 ± 13.5 (P = 0.043) for the Gross Motor Function Measure, reflecting subjects’ decline in motor function on examination and by report. These standardized assessments of clinical function are the first to be validated in giant axonal neuropathy and will be used in an upcoming gene therapy clinical trial.
- Subjects
FRIEDREICH'S ataxia; CEREBELLUM degeneration; NEUROPATHY; NEUROLOGICAL disorders; NERVOUS system abnormalities
- Publication
Journal of Child Neurology, 2015, Vol 30, Issue 6, p741
- ISSN
0883-0738
- Publication type
Article
- DOI
10.1177/0883073814542946