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Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial.
Köhler, Wolfgang; Engelen, Marc; Eichler, Florian; Lachmann, Robin; Fatemi, Ali; Sampson, Jacinda; Salsano, Ettore; Gamez, Josep; Molnar, Maria Judit; Pascual, Sílvia; Rovira, Maria; Vilà, Anna; Pina, Guillem; Martín-Ugarte, Itziar; Mantilla, Adriana; Pizcueta, Pilar; Rodríguez-Pascau, Laura; Traver, Estefania; Vilalta, Anna; Pascual, María; Martinell, Marc; Meya, Uwe; Mochel, Fanny.
Afiliación
  • Köhler W; Department of Neurology, University of Leipzig Medical Center, Leipzig, Germany. Electronic address: wolfgang.koehler@medizin.uni-leipzig.de.
  • Engelen M; Department of Pediatric Neurology, Amsterdam University Medical Centers, Amsterdam, Netherlands.
  • Eichler F; Department of Neurology, Harvard Medical School, Boston, MA, USA.
  • Lachmann R; Charles Dent Metabolic Unit, University College London Hospitals, London, UK.
  • Fatemi A; Department of Neurology and Developmental Medicine, Kennedy Krieger Institute, Baltimore, MD, USA.
  • Sampson J; Department of Neurology, Stanford University, Stanford, CA, USA.
  • Salsano E; Unit of Rare Neurodegenerative and Neurometabolic Diseases, Fondazione IRCCS Istituto Neurologico C Besta, Milan, Italy.
  • Gamez J; Department of Neurology, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
  • Molnar MJ; Institute of Genomic Medicine and Rare Disorders, Semmelweis University, Budapest, Hungary.
  • Pascual S; Minoryx Therapeutics, Barcelona, Spain.
  • Rovira M; Minoryx Therapeutics, Barcelona, Spain.
  • Vilà A; Minoryx Therapeutics, Barcelona, Spain.
  • Pina G; Minoryx Therapeutics, Barcelona, Spain.
  • Martín-Ugarte I; Minoryx Therapeutics, Barcelona, Spain.
  • Mantilla A; Minoryx Therapeutics, Barcelona, Spain.
  • Pizcueta P; Minoryx Therapeutics, Barcelona, Spain.
  • Rodríguez-Pascau L; Minoryx Therapeutics, Barcelona, Spain.
  • Traver E; Minoryx Therapeutics, Barcelona, Spain.
  • Vilalta A; Minoryx Therapeutics, Barcelona, Spain.
  • Pascual M; Minoryx Therapeutics, Barcelona, Spain.
  • Martinell M; Minoryx Therapeutics, Barcelona, Spain.
  • Meya U; Minoryx Therapeutics, Barcelona, Spain.
  • Mochel F; APHP-Brain and Spine Institute Department of Genetics, Reference Centre for Leukodystrophies, and Paris Brain Institute, Sorbonne University, La Pitié-Salpêtrière University Hospital, Paris, France.
Lancet Neurol ; 22(2): 127-136, 2023 02.
Article en En | MEDLINE | ID: mdl-36681445
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Adrenoleucodistrofia Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adult / Humans / Male País/Región como asunto: Europa Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2023 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Adrenoleucodistrofia Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adult / Humans / Male País/Región como asunto: Europa Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2023 Tipo del documento: Article