Efficacy and Safety of Leriglitazone in Patients With Friedreich Ataxia: A Phase 2 Double-Blind, Randomized Controlled Trial (FRAMES)

BACKGROUND AND OBJECTIVES: Friedreich ataxia (FRDA) is an autosomal recessive ataxia with no approved treatments. Leriglitazone is a selective peroxisome proliferator–activated receptor γ agonist that crosses the blood-brain barrier and, in preclinical models, improved mitochondrial function and ene...

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Detalles Bibliográficos
Autores principales: Pandolfo, Massimo, Reetz, Kathrin, Darling, Alejandra, Rodriguez de Rivera, Francisco Javier, Henry, Pierre-Gilles, Joers, James, Lenglet, Christophe, Adanyeguh, Isaac, Deelchand, Dinesh, Mochel, Fanny, Pousset, Françoise, Pascual, Sílvia, Van den Eede, Delphine, Martin-Ugarte, Itziar, Vilà-Brau, Anna, Mantilla, Adriana, Pascual, María, Martinell, Marc, Meya, Uwe, Durr, Alexandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Wolters Kluwer 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9747094/
https://www.ncbi.nlm.nih.gov/pubmed/36524101
http://dx.doi.org/10.1212/NXG.0000000000200034

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