ASPIRO Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Efficacy and Safety Findings - Blaschek, A.; Shieh, P. B.; Kuntz, N.; Dowling, J. J.; Müller-Felber, W.; Bönnemann, C. G.; Foley, A. R.; Saade, D. N.; Seferian, A. M.; Servais, L.; Lawlor, M. W.; Noursalehi, M.; Prasad, S.; Rico, S.; Miller, W. (Neuropediatrics, 2021)