Effects of Gene Replacement Therapy with Resamirigene Bilparvovec (AT132) on Skeletal Muscle Pathology in X-linked Myotubular Myopathy: Results from a Substudy of the ASPIRO Open-Label Clinical Trial
EBIOMEDICINE（2024）
关键词
X-linked myotubular myopathy,Centronuclear myopathy,Gene replacement therapy,Adeno-associated viral vector,Pathology,Human
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