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依达拉奉作为肌萎缩侧索硬化症患者治疗选择的作用。

Role of Edaravone as a Treatment Option for Patients with Amyotrophic Lateral Sclerosis.

作者信息

Cho HaEun, Shukla Surabhi

机构信息

College of Pharmacy, Larkin University, 18301 N Miami Ave, Miami, FL 33169, USA.

出版信息

Pharmaceuticals (Basel). 2020 Dec 31;14(1):29. doi: 10.3390/ph14010029.

Abstract

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease, is a progressive and fatal neurodegenerative disease that leads to a loss of muscle control due to nerve cells being affected in the brain and spinal cord. Some of the common clinical presentations of ALS include weakness of muscles, changes in behavior, dysfunction in speech, and cognitive difficulties. The cause of ALS is uncertain, but through several studies, it is known that mutations in SOD1 or C9orf72 genes could play a role as a factor of ALS. In addition, studies indicate that an excessive amount of free radicals, the reactive oxygen species (ROS), leads to neuronal damage by the peroxidation of unsaturated fatty acids in the neuronal cells. Edaravone, the newly approved antioxidant drug for ALS, halts the progression of ALS in the early stages through its cytoprotective effect and protects the nerves by reducing ROS. In this review, different aspects of ALS will be discussed, including its pathology, genetic aspect, and diagnosis. This review also focuses on edaravone as a treatment option for ALS, its mechanism of action, and its pharmacological properties. Clinical trials and adverse effects of edaravone and care for ALS patient are also discussed.

摘要

肌萎缩侧索硬化症(ALS),也被称为卢伽雷氏病,是一种进行性致命的神经退行性疾病,由于大脑和脊髓中的神经细胞受到影响,导致肌肉控制能力丧失。ALS的一些常见临床表现包括肌肉无力、行为改变、言语功能障碍和认知困难。ALS的病因尚不确定,但通过多项研究可知,SOD1或C9orf72基因的突变可能是ALS的一个致病因素。此外,研究表明,过量的自由基,即活性氧(ROS),会通过神经元细胞中不饱和脂肪酸的过氧化作用导致神经元损伤。依达拉奉是新批准用于治疗ALS的抗氧化药物,它通过其细胞保护作用在早期阶段阻止ALS的进展,并通过减少ROS来保护神经。在这篇综述中,将讨论ALS的不同方面,包括其病理学、遗传学方面和诊断。本综述还重点关注依达拉奉作为ALS的一种治疗选择、其作用机制及其药理学特性。还讨论了依达拉奉的临床试验和不良反应以及对ALS患者的护理。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3942/7823603/fb7f56060c95/pharmaceuticals-14-00029-g001.jpg

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