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Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Henzi BC, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Putananickal N, Stimpson G; North Star Consortium; Amthor H, Childs AM, Deconinck N, de Groot I, Horrocks I, Houwen-van Opstal S, Laugel V, Lopez Lobato M, Madruga Garrido M, Nascimento Osorio A, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Henzi BC, et al. Among authors: rubino nacht d. Lancet Neurol. 2023 Oct;22(10):890-899. doi: 10.1016/S1474-4422(23)00285-5. Lancet Neurol. 2023. PMID: 37739572 Clinical Trial.
Implementation of Motor Function Measure score percentile curves - Predicting motor function loss in Duchenne muscular dystrophy.
Hafner P, Schmidt S, Schädelin S, Rippert P, Hamroun D, Fabien S, Henzi B, Putananickal N, Rubino-Nacht D, Vuillerot C, Fischer D; MFM registry Study Group. Hafner P, et al. Among authors: rubino nacht d. Eur J Paediatr Neurol. 2022 Jan;36:78-83. doi: 10.1016/j.ejpn.2021.11.004. Epub 2021 Dec 14. Eur J Paediatr Neurol. 2022. PMID: 34929615 Free article.
Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne muscular dystrophy: A cross-sectional analysis.
Schmidt S, Hafner P, Klein A, Rubino-Nacht D, Gocheva V, Schroeder J, Naduvilekoot Devasia A, Zuesli S, Bernert G, Laugel V, Bloetzer C, Steinlin M, Capone A, Gloor M, Tobler P, Haas T, Bieri O, Zumbrunn T, Fischer D, Bonati U. Schmidt S, et al. Among authors: rubino nacht d. Neuromuscul Disord. 2018 Jan;28(1):16-23. doi: 10.1016/j.nmd.2017.10.003. Epub 2017 Nov 21. Neuromuscul Disord. 2018. PMID: 29174526 Clinical Trial.