Safety and Efficacy of Gene Replacement Therapy for X-linked Myotubular Myopathy (ASPIRO): a Multinational, Open-Label, Dose-Escalation Trial
Perry B. Shieh,Nancy L. Kuntz, James J. Dowling,Wolfgang Mueller-Felber, Carsten G. Bonnemann,Andreea M. Seferian, Laurent Servais,Barbara K. Smith, Francesco Muntoni,Astrid Blaschek, A. Reghan Foley,Dimah N. Saade,Sarah Neuhaus,Lindsay N. Alfano, Alan H. Beggs,Ana Buj-Bello, Martin K. Childers, Tina Duong, Robert J. Graham,Minal Jain,Julie Coats, Vicky Macbean,Emma S. James, Jun Lee, Fulvio Mavilio,Weston Miller,Fatbardha Varfaj,Michael Murtagh, Cong Han,Mojtaba Noursalehi, Michael W. Lawlor,Suyash Prasad,Salvador Rico LANCET NEUROLOGY（2023）
Key words
Fragile X Syndrome
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