Dr Perry B Shieh, from the David Geffen School of Medicine at the University of California in Los Angeles, presents the findings of his trial published in The Lancet Neurology on Nov 15, 2023: Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
Read the full paper, "Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial", here:
www.thelancet.com/journals/la...
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15 ноя 2023
