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rohrer am
(3 results)?
Mortality of symptomatic children with spinal muscular atrophy in the era of disease-modifying therapies.
Neuromuscul Disord. 2025 Apr;49:105313. doi: 10.1016/j.nmd.2025.105313. Epub 2025 Feb 17.
Neuromuscul Disord. 2025.
PMID: 40022865
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.
Lancet Neurol. 2023 Oct;22(10):890-899. doi: 10.1016/S1474-4422(23)00285-5.
Lancet Neurol. 2023.
PMID: 37739572
Clinical Trial.
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