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Adaptive behavior assessed by Vineland-3 as comprehensive outcome measure in vanishing white matter.
Ann Clin Transl Neurol. 2024 Mar;11(3):650-661. doi: 10.1002/acn3.51985. Epub 2024 Jan 12.
Ann Clin Transl Neurol. 2024.
PMID: 38217081
Free PMC article.
International validation of meaningfulness of postural sway and gait to assess myeloneuropathy in adults with adrenoleukodystrophy.
Yska HAF, Turk BR, Fatemi A, Goodman J, Voermans M, Amos D, Amanat M, van de Stadt S, Engelen M, Smith-Fine A, Keller J.
Yska HAF, et al.
J Inherit Metab Dis. 2024 May 25. doi: 10.1002/jimd.12753. Online ahead of print.
J Inherit Metab Dis. 2024.
PMID: 38795020
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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 W, Engelen M, Eichler F, Lachmann R, Fatemi A, Sampson J, Salsano E, Gamez J, Molnar MJ, Pascual S, Rovira M, Vilà A, Pina G, Martín-Ugarte I, Mantilla A, Pizcueta P, Rodríguez-Pascau L, Traver E, Vilalta A, Pascual M, Martinell M, Meya U, Mochel F; ADVANCE Study Group.
Köhler W, et al.
Lancet Neurol. 2023 Feb;22(2):127-136. doi: 10.1016/S1474-4422(22)00495-1.
Lancet Neurol. 2023.
PMID: 36681445
Clinical Trial.
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