围绕脊髓性肌萎缩症基因和遗传疗法的商业用途进行临床决策。

IF 8.3 2区材料科学 Q1 MATERIALS SCIENCE, MULTIDISCIPLINARY

ACS Applied Materials & Interfaces Pub Date : 2024-07-01 DOI:10.1016/j.neurot.2024.e00437

Megan A. Waldrop

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引用次数: 0

摘要

脊髓性肌萎缩症不再是美国婴儿遗传性死亡的主要原因。自2016年以来，已有三种基因疗法获准用于治疗脊髓性肌萎缩症。每种疗法都经过了充分的研究，在安全性和有效性方面都有可靠的数据。然而，目前还没有头对头的比较研究为临床决策提供依据。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自然病史的不断变化，我们需要更多的数据来帮助我们做出以证据为基础、具有成本效益的临床决策。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Clinical decision making around commercial use of gene and genetic therapies for spinal muscular atrophy

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.

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来源期刊

ACS Applied Materials & Interfaces 工程技术-材料科学：综合

CiteScore

16.00

自引率

6.30%

发文量

4978

审稿时长

1.8 months

期刊介绍： ACS Applied Materials & Interfaces is a leading interdisciplinary journal that brings together chemists, engineers, physicists, and biologists to explore the development and utilization of newly-discovered materials and interfacial processes for specific applications. Our journal has experienced remarkable growth since its establishment in 2009, both in terms of the number of articles published and the impact of the research showcased. We are proud to foster a truly global community, with the majority of published articles originating from outside the United States, reflecting the rapid growth of applied research worldwide.