Abstract Spinal muscular atrophy (SMA) used to be one of the most common genetic causes of infant mortality. New disease modifying treatments have changed the disease trajectories and most impressive results are seen if treatment is initiated in the presymptomatic phase of the disease. Very recently, the European Medicine Agency approved Onasemnogene abeparvovec (Zolgensma®) for the treatment of patients with SMA with up to three copies of the SMN2 gene or the clinical presentation of SMA type 1. While this broad indication provides new opportunities, it also triggers discussions on the appropriate selection of patients in the context of limited available evidence. To aid the rational use of Onasemnogene abeparvovec for the treatment of SMA, a group of European neuromuscular experts presents in this paper eleven consensus statements covering qualification, patient selection, safety considerations and long-term monitoring.

中文翻译：

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欧洲关于脊髓性肌萎缩症基因替代疗法的特别共识声明

摘要 脊髓性肌萎缩症 (SMA) 曾经是导致婴儿死亡的最常见遗传原因之一。新的疾病改善治疗改变了疾病的发展轨迹，如果在疾病的症状前阶段开始治疗，就会看到最令人印象深刻的结果。最近，欧洲药品管理局批准 Onasemnogene abeparvovec (Zolgensma®) 用于治疗具有多达三个 SMN2 基因拷贝或 1 型 SMA 临床表现的 SMA 患者。 虽然这一广泛的适应症提供了新的机会，但它也触发了discussions on the appropriate selection of patients in the context of limited available evidence. 为了帮助合理使用 Onasemnogene abeparvovec 治疗 SMA，