机译:SMA1的AVXS-101(OnAsmaNogene AbepeRvovec):比较研究与预期自然历史队列
Center for Gene Therapy Research Institute Nationwide Children’s Hospital;
Department of Neurology The Ohio State University Wexner Medical Center;
Center for Gene Therapy Research Institute Nationwide Children’s Hospital;
Center for Gene Therapy Research Institute Nationwide Children’s Hospital;
Department of Pediatrics The Ohio State University Wexner Medical Center;
Center for Gene Therapy Research Institute Nationwide Children’s Hospital;
AveXis Inc;
AveXis Inc;
AveXis Inc;
AveXis Inc;
AveXis Inc;
AveXis Inc;
AveXis Inc;
Department of Neurology The Ohio State University Wexner Medical Center;
Department of Pediatrics The Ohio State University Wexner Medical Center;
AveXis Inc;
Center for Gene Therapy Research Institute Nationwide Children’s Hospital;
Gene therapy; spinal muscular atrophy; natural history; neuromuscular diseases; ClinicalTrials.gov NCT02122952 (https://clinicaltrials.gov/ct2/show/NCT02122952) and NCT01736553(https://clinicaltrials.gov/ct2/show/NCT01736553);
机译:SMA1的AVXS-101(OnAsmaNogene AbepeRvovec):比较研究与预期自然历史队列
机译:无活动的生存和电机里程碑取得诸如OnAsmaNogene Abeparvovec和Nusinersen干预措施对比脊髓肌肉萎缩T1(SMA1)患者的自然历史形成对比
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