

Initial studies have indicated that these measures are feasible, reliable and valid. The primary aims of ACT-CMT include validation of the Charcot-Marie-Tooth Functional Outcome Measure, magnetic resonance imaging of intramuscular fat accumulation as a lower limb motor biomarker, and in-vivo reflectance confocal microscopy of Meissner corpuscle sensory receptor density, a sensory biomarker. Accelerate Clinical Trials in CMT (ACT-CMT) is an international study designed to address important gaps in CMT1A clinical trial readiness including the lack of a validated, responsive functional outcome measure for adults, and a lack of validated biomarkers for multicenter application in clinical trials in CMT1A. With therapeutic trials on the horizon for Charcot-Marie-Tooth type 1A (CMT1A), reliable, valid, and responsive clinical outcome assessments and biomarkers are essential. 8Department of Neurology, Carver College of Medicine, University of Iowa, Iowa City, IA, United States.7Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, United States.6Department of Clinical Neurosciences, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.5Centre for Neuromuscular Diseases, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, United Kingdom.



Herrmann 1 on behalf of The ACT-CMT Study Group McDermott 1,3 Jeffrey Krischer 4 John Thornton 5 Davide Pareyson 6 Steven S.
