Clinical trials in Charcot-Marie-Tooth disorders: a retrospective and preclinical assessment
Malavika A. Nair,
Zhiyv Niu,
Nicholas N. Madigan
et al.
Abstract:ObjectiveThis study aimed to evaluate the progression of clinical and preclinical trials in Charcot-Marie-Tooth (CMT) disorders.BackgroundCMT has historically been managed symptomatically and with genetic counseling. The evolution of molecular and pathologic understanding holds a therapeutic promise in gene-targeted therapies.MethodsClinicalTrials.gov from December 1999 to June 2022 was data extracted for CMT with preclinical animal gene therapy trials also reviewed by PubMed search.ResultsThe number of active… Show more
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