Abstract：
Spinal muscular atrophy is a rare and progressive neuromuscular disease that, without treatment, leads to progressive weakness and often death. A plethora of studies have led to the approval of three high-cost and effective treatments since 2016. These treatments, nusinersen, onasemnogene abeparvovec, and risdiplam, have not been directly compared and have varying challenges in administration. In this review, we discuss the evidence supporting the use of these medications, the process of treatment selection, monitoring after treatment, the limited data comparing treatments, as well as future directions for investigation and therapy.
摘要：
脊髓性肌萎缩症是一种罕见的进行性神经肌肉疾病,如果没有治疗,导致渐进的虚弱和经常死亡。自2016年以来，大量研究导致批准了三种高成本和有效的治疗方法。这些治疗,Nusinersen,前基因abeparvovec和risdiplam，没有直接比较，在管理方面也有不同的挑战。在这次审查中,我们讨论支持使用这些药物的证据，治疗选择的过程，治疗后的监测，有限的数据比较治疗，以及未来的研究和治疗方向。
