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The Lancet. Neurology|November 17, 2023
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trialPerry B Shieh, Nancy L Kuntz, James J Dowling, et al.Journal of Neuromuscular Diseases|June 13, 2022
INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular MyopathyJames J Dowling, Wolfgang Müller-Felber, Barbara K Smith, et al.Ebiomedicine|December 12, 2023
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trialMichael W Lawlor, Benedikt Schoser, Marta Margeta, et al.Pageof 6