Tofersen 用于 SOD1 ALS。

IF 2.3 Q3 CLINICAL NEUROLOGY Neurodegenerative disease management Pub Date : 2024-01-01 Epub Date: 2024-09-27 DOI:10.1080/17582024.2024.2402216
William H Everett, Robert C Bucelli
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引用次数: 0

摘要

肌萎缩性脊髓侧索硬化症(ALS)是一种影响运动系统的神经退行性疾病。ALS 的异质性使试验设计变得复杂。ALS 的遗传形式为在异质性较低的人群中进行干预提供了机会。肌萎缩性脊髓侧索硬化症与 SOD1 的功能增益突变有关,因此 "基因敲除 "策略是一种很有吸引力的治疗方法。Tofersen是一种反义寡核苷酸,可通过RNA酶介导的SOD1 mRNA降解减少SOD1的表达。虽然托非森的 III 期试验未能达到主要终点,但开放标签扩展数据表明,托非森可延缓 SOD1 ALS 的进展。
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Tofersen for SOD1 ALS.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative condition affecting the motor system. The heterogenous nature of ALS complicates trial design. Genetic forms of ALS present an opportunity to intervene in a less heterogeneous population. ALS associated with gain of function mutations in SOD1 make 'knock-down' strategies an attractive therapeutic approach. Tofersen, an antisense oligonucleotide that reduces expression of SOD1 via RNAase mediated degradation of SOD1 mRNA, has shown robust effects on ALS biomarkers. While a Phase III trial of tofersen failed to meet its primary end point, open label extension data suggests that tofersen slows progression of SOD1 ALS.

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