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...
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 |
Similar Items
-
Tolerability and efficacy study of P2X7 inhibition in experimental Charcot-Marie-Tooth type 1A (CMT1A) neuropathy
by: Giovanna Sociali, et al.
Published: (2016-11-01) -
Schwann cell differentiation in Charcot-Marie-Tooth disease 1 A (CMT1A)
by: Abdelaal, Tamer
Published: (2019) -
Unmasking a Case of Asymptomatic Charcot-Marie-Tooth Disease (CMT1A) With Vincristine
by: Roopam Jariwal MS, et al.
Published: (2018-02-01) -
Genetics of Charcot-Marie-Tooth (CMT) Disease within the Frame of the Human Genome Project Success
by: Vincent Timmerman, et al.
Published: (2014-01-01) -
Pathogenic mechanism of the FIG4 mutation responsible for Charcot-Marie-Tooth disease CMT4J.
by: Guy M Lenk, et al.
Published: (2011-06-01)