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A natural history comparison of SOD1-mutant patients with amyotrophic lateral sclerosis between Chinese and German populations.

作者信息

Tang Lu, Dorst Johannes, Chen Lu, Liu Xiaolu, Ma Yan, Günther Kornelia, Michels Sebastian, Müller Kathrin, Freischmidt Axel, Weishaupt Jochen H, Fan Dongsheng, Ludolph Albert C

机构信息

Department of Neurology, Peking University Third Hospital, Beijing, 100191, China.

Beijing Key Laboratory of Biomarker and Translational Research in Neurodegenerative Diseases, Peking University Third Hospital, Beijing, 100191, China.

出版信息

Transl Neurodegener. 2021 Oct 28;10(1):42. doi: 10.1186/s40035-021-00266-x.

DOI:10.1186/s40035-021-00266-x
PMID:34711284
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8555265/
Abstract
摘要

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本文引用的文献

1
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for ALS.针对肌萎缩性侧索硬化症的反义寡核苷酸 Tofersen 的 1-2 期临床试验。
N Engl J Med. 2020 Jul 9;383(2):109-119. doi: 10.1056/NEJMoa2003715.
2
Prognostic factors in ALS: a comparison between Germany and China.肌萎缩侧索硬化症的预后因素:中德之间的比较。
J Neurol. 2019 Jun;266(6):1516-1525. doi: 10.1007/s00415-019-09290-4. Epub 2019 Mar 28.
3
Better survival in female -mutant patients with ALS: a study of -related natural history.肌萎缩侧索硬化症女性突变患者的更好生存率:一项相关自然史研究。
Transl Neurodegener. 2019 Jan 8;8:2. doi: 10.1186/s40035-018-0142-8. eCollection 2019.
4
Identification of an A4V SOD1 mutation in a Chinese patient with amyotrophic lateral sclerosis without the A4V founder effect common in North America.在一名无北美常见A4V奠基者效应的中国肌萎缩侧索硬化症患者中鉴定出A4V SOD1突变。
Amyotroph Lateral Scler Frontotemporal Degener. 2018 Aug;19(5-6):466-468. doi: 10.1080/21678421.2018.1451895. Epub 2018 Mar 22.
5
Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial.依达拉奉在明确诊断的肌萎缩侧索硬化症患者中的安全性和有效性:一项随机、双盲、安慰剂对照试验。
Lancet Neurol. 2017 Jul;16(7):505-512. doi: 10.1016/S1474-4422(17)30115-1. Epub 2017 May 15.
6
ALS Clinical Trials Review: 20 Years of Failure. Are We Any Closer to Registering a New Treatment?肌萎缩侧索硬化症临床试验综述:二十年的失败。我们距离批准一种新疗法更近了吗?
Front Aging Neurosci. 2017 Mar 22;9:68. doi: 10.3389/fnagi.2017.00068. eCollection 2017.
7
Genetic epidemiology of amyotrophic lateral sclerosis: a systematic review and meta-analysis.肌萎缩侧索硬化症的遗传流行病学:系统评价和荟萃分析。
J Neurol Neurosurg Psychiatry. 2017 Jul;88(7):540-549. doi: 10.1136/jnnp-2016-315018. Epub 2017 Jan 5.
8
Defining SOD1 ALS natural history to guide therapeutic clinical trial design.定义超氧化物歧化酶1型肌萎缩侧索硬化症的自然史以指导治疗性临床试验设计。
J Neurol Neurosurg Psychiatry. 2017 Feb;88(2):99-105. doi: 10.1136/jnnp-2016-313521. Epub 2016 Jun 3.
9
Natural history of young-adult amyotrophic lateral sclerosis.青年型肌萎缩侧索硬化症的自然病史。
Neurology. 2008 Sep 16;71(12):876-81. doi: 10.1212/01.wnl.0000312378.94737.45. Epub 2008 Jul 2.