Charcot Marie Tooth Disease Clinical Trial
Official title:
Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease
It is necessary for outcome measures to accurately reflect the state of health of a person in order for clinical trials to show benefit. The most commonly used outcome measure for Charcot Marie Tooth Disease (CMT) is the CMT Neuropathy Score, which uses cutoffs of points designated as mild (0-10 points), moderate (11-20) or severe (21-36). These terms are arbitrary. This study is looking to base mild, moderate, and severe on what both people affected with CMT and those who provide for people with CMT consider appropriate.
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Observational Model: Cohort, Time Perspective: Prospective
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