Measuring Disease Progression in Giant Axonal Neuropathy: Implications for Clinical Trial Design

Measuring Disease Progression in Giant Axonal Neuropathy: Implications for Clinical Trial Design ; Lisa A. Roth, MD, MS,
Jonathan D. Marra, MA, Nicole H. LaMarca, DNP, MSN, CPNCJ; Child Neurol May 2015 30: 741-748 doi: 10.1177/0883073814542946

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 ....