My trial and training journey in X-linked myotubular myopathy: mountains and valleys
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Publisher
Elsevier BV
Reference8 articles.
1. Characterization of mutations in the myotubularin gene in twenty-six patients with X-linked myotubular myopathy;de Gouyon;Hum Mol Genet,1997
2. Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial;Shieh;Lancet Neurol,2023
3. An integrated modelling methodology for estimating the prevalence of centronuclear myopathy;Vandersmissen;Neuromuscul Disord,2018
4. Aerobic Training in Patients with Congenital Myopathy;Hedermann;PLoS One,2016
5. all participants;Pater;Neuromuscul Disord,2023
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