Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A (CMT1A).

Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A (CMT1A).

The most common type of Charcot-Marie-Tooth disease is caused by a duplication of PMP22 leading to dysmyelination, axonal loss and progressive muscle weakness (CMT1A). Currently, no approved therapy is available for CMT1A patients. A novel polytherapeutic proof-of-principle approach using PXT3003, a...

Full description

Bibliographic Details
Main Authors: Thomas Prukop, Jan Stenzel, Stephanie Wernick, Theresa Kungl, Magdalena Mroczek, Julia Adam, David Ewers, Serguei Nabirotchkin, Klaus-Armin Nave, Rodolphe Hajj, Daniel Cohen, Michael W Sereda
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2019-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0209752

Internet

https://doi.org/10.1371/journal.pone.0209752

Similar Items