A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A

BACKGROUND: Charcot–Marie–Tooth disease type 1A (CMT1A) is a rare, orphan, hereditary neuromuscular disorder with no cure and for which only symptomatic treatment is currently available. A previous phase 2 trial has shown preliminary evidence of efficacy for PXT3003 in treating CMT1A. This phase 3,...

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Detalles Bibliográficos
Autores principales: Attarian, Shahram, Young, Peter, Brannagan, Thomas H., Adams, David, Van Damme, Philip, Thomas, Florian P., Casanovas, Carlos, Tard, Céline, Walter, Maggie C., Péréon, Yann, Walk, David, Stino, Amro, de Visser, Marianne, Verhamme, Camiel, Amato, Anthony, Carter, Gregory, Magy, Laurent, Statland, Jeffrey M., Felice, Kevin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8520617/
https://www.ncbi.nlm.nih.gov/pubmed/34656144
http://dx.doi.org/10.1186/s13023-021-02040-8

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