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2014年美国肌萎缩侧索硬化症患病率

Prevalence of Amyotrophic Lateral Sclerosis - United States, 2014.

作者信息

Mehta Paul, Kaye Wendy, Raymond Jaime, Wu Ruoming, Larson Theodore, Punjani Reshma, Heller Daniel, Cohen Jessica, Peters Tracy, Muravov Oleg, Horton Kevin

机构信息

Division of Toxicology and Human Health Sciences, Agency for Toxic Substances and Disease Registry, CDC.

出版信息

MMWR Morb Mortal Wkly Rep. 2018 Feb 23;67(7):216-218. doi: 10.15585/mmwr.mm6707a3.

DOI:10.15585/mmwr.mm6707a3
PMID:29470458
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5858037/
Abstract

Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease, is a progressive and fatal neuromuscular disease; the majority of ALS patients die within 2-5 years of receiving a diagnosis (1). Familial ALS, a hereditary form of the disease, accounts for 5%-10% of cases, whereas the remaining sporadic cases have no clearly defined etiology (1). ALS affects persons of all races and ethnicities; however, whites, males, non-Hispanics, persons aged >60 years, and those with a family history of ALS are more likely to develop the disease (1-3). No cure for ALS has yet been identified, and the lack of proven and effective therapeutic interventions is an ongoing challenge. Current treatments available do not cure ALS but have been shown to slow disease progression. Until recently, only one drug (riluzole) was approved to treat ALS; however, in 2017, the Food and Drug Administration approved a second drug, edaravone (4).

摘要

肌萎缩侧索硬化症(ALS),通常被称为卢伽雷氏病,是一种进行性的致命神经肌肉疾病;大多数ALS患者在确诊后2至5年内死亡(1)。家族性ALS是该疾病的一种遗传形式,占病例的5%至10%,而其余的散发性病例没有明确的病因(1)。ALS影响所有种族和族裔的人群;然而,白人、男性、非西班牙裔、60岁以上的人以及有ALS家族史的人更易患此病(1 - 3)。目前尚未找到治愈ALS的方法,缺乏经过验证的有效治疗干预措施仍是一个持续存在的挑战。现有的治疗方法无法治愈ALS,但已证明可减缓疾病进展。直到最近,只有一种药物(利鲁唑)被批准用于治疗ALS;然而,在2017年,美国食品药品监督管理局批准了第二种药物,依达拉奉(4)。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/59a8/5858037/5f4e8684226d/mm6707a3-F.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/59a8/5858037/5f4e8684226d/mm6707a3-F.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/59a8/5858037/5f4e8684226d/mm6707a3-F.jpg

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本文引用的文献

1
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MMWR Surveill Summ. 2016 Aug 5;65(8):1-12. doi: 10.15585/mmwr.ss6508a1.
2
Quantifying a nonnotifiable disease in the United States: the National Amyotrophic Lateral Sclerosis Registry model.在美国对一种非法定报告疾病进行量化:国家肌萎缩侧索硬化症登记模型。
JAMA. 2014 Sep 17;312(11):1097-8. doi: 10.1001/jama.2014.9799.
3
Prevalence of amyotrophic lateral sclerosis - United States, 2010-2011.2010 - 2011年美国肌萎缩侧索硬化症的患病率
肌萎缩侧索硬化症的发病率和死亡率:一项基于全国人口的42年研究。
J Neurol. 2024 Dec 12;272(1):44. doi: 10.1007/s00415-024-12743-0.
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Serine metabolism in aging and age-related diseases.衰老及与年龄相关疾病中的丝氨酸代谢
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Neurofilament light chain: a biomarker at the crossroads of clarity and confusion for gene-directed therapies.神经丝轻链:一种处于基因导向治疗的清晰与困惑交叉点的生物标志物。
Neurodegener Dis Manag. 2024;14(6):227-239. doi: 10.1080/17582024.2024.2421738. Epub 2024 Nov 15.
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Comparison of Demographics: National Amyotrophic Lateral Sclerosis Registry and Clinical Trials Data.人口统计学比较:国家肌萎缩侧索硬化症注册库与临床试验数据
J Racial Ethn Health Disparities. 2024 Jul 8. doi: 10.1007/s40615-024-02047-4.
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Enhancing Clinical Infrastructure for the Delivery of Intrathecal and Genetic Therapies: A Qalsody (Tofersen) Model for Patients With -ALS.加强鞘内注射和基因治疗的临床基础设施:肌萎缩侧索硬化症患者的Qalsody(托弗生)模式。
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Medicina (Kaunas). 2023 Sep 29;59(10):1745. doi: 10.3390/medicina59101745.
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