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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Detalhes bibliográficos
Publicado no:Neurol Res Pract
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2019
Assuntos:
Clinical Trial Protocol
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7650055/
https://ncbi.nlm.nih.gov/pubmed/33324899
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s42466-019-0038-9
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