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Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.
McDonald CM, Guglieri M, Vučinić D, Acsadi G, Brandsema JF, Bruno C, Finanger EL, Harper A, Lobato ML, Masson R, Muelas N, Munell F, Nevo Y, Péréon Y, Phan H, Sansone VA, Scoto M, Willis T, Finkel RS, Vandenborne K, Cazzaniga S, Montrasio S, Alessi F, Bettica P, Mercuri E; Givinostat Study 51 Investigators; Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS) Investigators; ImagingDMD Investigators. McDonald CM, et al. Among authors: lobato ml. Ann Clin Transl Neurol. 2025 Nov;12(11):2335-2348. doi: 10.1002/acn3.70165. Epub 2025 Aug 19. Ann Clin Transl Neurol. 2025. PMID: 40830818 Free PMC article. Clinical Trial.
Safety and efficacy of tamoxifen in non-ambulant patients with Duchenne muscular dystrophy: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial (TAMDMD Group B).
Henzi BC, Putananickal N, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Amthor H, Childs AM, Deconinck N, Horrocks I, Houwen-van Opstal S, Laugel V, Lobato ML, Osorio AN, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Henzi BC, et al. Among authors: lobato ml. Neuromuscul Disord. 2025 Feb;47:105275. doi: 10.1016/j.nmd.2025.105275. Epub 2025 Jan 16. Neuromuscul Disord. 2025. PMID: 39879732 Free article. Clinical Trial.