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托弗森:美好的一面还是夸张? 这就是为什么我喜欢它

Tanushree Chawla1, Vinay Goyal1

  • 1Department of Neurology, Institute of Neurosciences, Medanta, The Medicity, Gurugram, Haryana, India.

Annals of Indian Academy of Neurology
|November 29, 2023
PubMed
概括

托弗森是第一个针对SOD1基因突变的被批准用于肌缩侧面硬化症 (ALS) 的基因疗法. 虽然它有效降低了疾病生物标志物,但临床改善在统计学上并不显著.

关键词:
肌缩性侧面硬化症 (AMLS) 是一种疾病.与SOD1相关的ALS.托弗森的意思是说.基因治疗的基因疗法

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