Rationale and protocol of a double-blind, randomized, placebo-controlled trial to test the efficacy, safety, and tolerability of dimethyl fumarate in Friedreich Ataxia (DMF-FA-201)

INTRODUCTION: Friedreich Ataxia (FRDA) is an autosomal recessive neurodegenerative disorder that causes gait and limb ataxia, dysarthria, and impaired vibratory sense, with cardiomyopathy being the predominant cause of death. There is no approved therapy, which results in the use of symptomatic trea...

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Detalles Bibliográficos
Autores principales: Pane, Chiara, Marra, Alberto Maria, Aliberti, Ludovica, Campanile, Mario, Coscetta, Federica, Crisci, Giulia, D'Assante, Roberta, Marsili, Angela, Puorro, Giorgia, Salzano, Andrea, Cittadini, Antonio, Saccà, Francesco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10513368/
https://www.ncbi.nlm.nih.gov/pubmed/37746147
http://dx.doi.org/10.3389/fnins.2023.1260977

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10513368/
https://www.ncbi.nlm.nih.gov/pubmed/37746147
http://dx.doi.org/10.3389/fnins.2023.1260977

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