Schwann cell transcript biomarkers for hereditary neuropathy skin biopsies.
John SvarenJohn J MoranXingyao WuRiccardo ZuccarinoChelsea BaconYunhong BaiRaghu RameshLaurie GutmannDaniel M AndersonDerek PavelecMichael E ShyPublished in: Annals of neurology (2019)
This platform should be useful in clinical trials for CMT1A as a biomarker of target engagement that can be used to optimize dosing, and the same normalization framework is applicable to other types of CMT. ANN NEUROL 2019;85:887-898.