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A natural history comparison Check for updates of SOD1-mutant patients with amyotrophic lateral sclerosis between Chinese and German populations 被引量:1

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摘要 Currently,there is no effective treatment for amyotrophic lateral sclerosis(ALS),despite the limited efficacy of riluzole[1]and edaravone[2].SOD1(coding for the Cu/Zn superoxide dismutase)is the second most frequent genetic cause for ALS only after C9orf72 in patients with European ancestry while being the most frequent in Asian ALS populations[3],Multiple therapeutic approaches have targeted SOD1-related ALS,including the antisense oligonucleotide tofersen with promising results in a recent phase I/II trial[4].
出处 《Translational Neurodegeneration》 SCIE CAS 2021年第4期551-553,共3页 转化神经变性病(英文)
基金 supported by grants from the National Natural Science Foundation of China(81873784,82071426 and 81901298) Clinical Cohort Construction Program of Peking University Third Hospital(BYSYDL2019002).
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