Protocol of a randomized, double-blind, placebo-controlled, parallel-group, multicentre study of the efficacy and safety of nicotinamide in patients with Friedreich ataxia (NICOFA)

INTRODUCTION: Currently, no treatment that delays with the progression of Friedreich ataxia is available. In the majority of patients Friedreich ataxia is caused by homozygous pathological expansion of GAA repeats in the first intron of the FXN gene. Nicotinamide acts as a histone deacetylase inhibi...

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Detalles Bibliográficos
Autores principales: Reetz, Kathrin, Hilgers, Ralf-Dieter, Isfort, Susanne, Dohmen, Marc, Didszun, Claire, Fedosov, Kathrin, Kistermann, Jennifer, Mariotti, Caterina, Durr, Alexandra, Boesch, Sylvia, Klopstock, Thomas, Rodríguez de Rivera Garrido, Francisco Javier, Schöls, Ludger, Klockgether, Thomas, Pandolfo, Massimo, Korinthenberg, Rudolf, Lavin, Philip, Molenberghs, Geert, Libri, Vincenzo, Giunti, Paola, Festenstein, Richard, Schulz, Jörg B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Clinical Trial Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7650055/
https://www.ncbi.nlm.nih.gov/pubmed/33324899
http://dx.doi.org/10.1186/s42466-019-0038-9

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