肌萎缩侧索硬化
SOD1
超氧化物歧化酶
寡核苷酸
突变
医学
基因
分子生物学
生物
遗传学
内科学
疾病
氧化应激
标识
DOI:10.58347/tml.2023.1681a
摘要
Tofersen (Qalsody – Biogen), an intrathecally administered antisense oligonucleotide, has received accelerated approval from the FDA for treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.
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