X-linked myotubular myopathy: A prospective international natural history study

<p><strong>Objectives:</strong><br /> Because X-linked myotubular myopathy (XLMTM) is a rare neuromuscular disease caused by mutations in the MTM1 gene with a large phenotypic heterogeneity, to ensure clinical trial readiness, it was mandatory to better quantify disease burde...

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Bibliographic Details
Main Authors:	Servais, L, Annoussamy, M, Lilien, C, Gidaro, T, Gargaun, E, Chê, V, Schara, U, Gangfuß, A, D'Amico, A, Dowling, JJ, Darras, BT, Daron, A, Hernandez, A, de Lattre, C, Arnal, J-M, Mayer, M, Cuisset, J-M, Vuillerot, C, Fontaine, S, Bellance, R, Biancalana, V, Buj-Bello, A, Hogrel, J-Y, Landy, H
Format:	Journal article
Language:	English
Published:	American Academy of Neurology 2019

X-linked myotubular myopathy: A prospective international natural history study

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