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...

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Detalles Bibliográficos
Autores principales: Prukop, Thomas, Stenzel, Jan, Wernick, Stephanie, Kungl, Theresa, Mroczek, Magdalena, Adam, Julia, Ewers, David, Nabirotchkin, Serguei, Nave, Klaus-Armin, Hajj, Rodolphe, Cohen, Daniel, Sereda, Michael W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6334894/
https://www.ncbi.nlm.nih.gov/pubmed/30650121
http://dx.doi.org/10.1371/journal.pone.0209752

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