Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA; Departments of Pediatrics and Neurology, Wexner Medical Center, Ohio State University, Columbus OH 43205, USA.
Neurotherapeutics. 2024 Jul;21(4):e00437. doi: 10.1016/j.neurot.2024.e00437. Epub 2024 Sep 5.
Spinal muscular atrophy is no longer a leading cause of inherited infant death in the United States. Since 2016, three genetic therapies have been approved for the treatment of spinal muscular atrophy. Each therapy has been well studied with robust data for both safety and efficacy. However, there are no head-to-head comparator studies to inform clinical decision making. Thus, treatment selection, timing, and combination therapy is largely up to clinician preference and insurance policies. As the natural history of spinal muscular atrophy continues to change, more data is needed to assist in evidence-based and cost-effective clinical decision making.
脊髓性肌萎缩症不再是美国导致婴儿遗传性死亡的主要原因。自 2016 年以来，已有三种基因疗法被批准用于治疗脊髓性肌萎缩症。每种疗法的安全性和有效性都有大量的研究数据支持。然而，目前还没有头对头的比较研究来为临床决策提供信息。因此，治疗的选择、时机和联合治疗在很大程度上取决于临床医生的偏好和保险政策。随着脊髓性肌萎缩症的自然病史不断变化，需要更多的数据来协助基于证据和具有成本效益的临床决策。
