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Cited article:

INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy

James J. Dowling, Wolfgang Müller-Felber, Barbara K. Smith, et al.
Journal of Neuromuscular Diseases 9 (4) 503 (2022)
https://doi.org/10.3233/JND-210781