Onasemnogene abeparvovec has been life-changing for children with spinal muscular atrophy (SMA), signifying the potential and progress occurring in gene- and cell-based therapies for rare genetic diseases. Hence, it is important that clinicians gain knowledge and understanding in gene therapy-based treatment strategies for SMA. In this review, we describe the development and translation of onasemnogene abeparvovec from clinical trials to healthcare practice and share knowledge on the facilitators and barriers to implementation. Rapid and accurate SMA diagnosis, awareness, and education to safely deliver gene therapy to eligible patients and access to expertise in multidisciplinary management for neuromuscular disorders are crucial for health system readiness. Early engagement and intersectoral collaboration are required to surmount complex logistical processes and develop policy, governance, and accountability. The collection and utilisation of real-world evidence are also an important part of clinical stewardship, informing ongoing improvements to care delivery and access. Additionally, a research-enabled clinical ecosystem can expand scientific knowledge and discovery to optimise future therapies and magnify health impacts. Important ethical, equity, economic, and sustainability issues are evident, for which we must connect globally.

中文翻译：

---

基于基因治疗的脊髓性肌萎缩症策略——亚太地区的视角

Onasemnogene abeparvovec 已经改变了脊髓性肌萎缩症 (SMA) 儿童的生活，标志着罕见遗传病的基因和细胞疗法的潜力和进展。因此，临床医生获得基于基因治疗的 SMA 治疗策略的知识和理解非常重要。在这篇综述中，我们描述了 onasemnogene abeparvovec 从临床试验到医疗保健实践的开发和转化，并分享了实施过程中的促进因素和障碍的知识。快速、准确的 SMA 诊断、认识和教育，以安全地为符合条件的患者提供基因治疗，并获得神经肌肉疾病多学科管理的专业知识，对于卫生系统的准备工作至关重要。需要早期参与和部门间协作来克服复杂的后勤流程并制定政策、治理和问责制。现实世界证据的收集和利用也是临床管理的重要组成部分，为护理服务的提供和获取的持续改进提供信息。此外，以研究为基础的临床生态系统可以扩展科学知识和发现，以优化未来的疗法并扩大健康影响。重要的道德、公平、经济和可持续发展问题显而易见，为此我们必须在全球范围内建立联系。