Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
2023
Shieh PB et Al.
Lancet neurology 22, 1125-1139. 26.
2023
Shieh PB et Al.
Lancet neurology 22, 1125-1139. 26.