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引用次数: 0
摘要
肌萎缩侧索硬化症是一种导致上下运动神经元丧失的神经退行性疾病。近 90% 的病例为散发性,其余为家族性。这种疾病的预后很差,美国食品和药物管理局(FDA)只批准了两种改变病情的药物。已获批准的治疗该疾病的药物对患者的生存益处非常有限。依达拉奉(Edaravone)是美国食品及药物管理局(FDA)批准的一种新药,可使部分 ALS 患者的病情恶化速度减慢 33%。本文介绍了可能对生存有益的各种干预措施,如早期诊断、药物治疗、基因治疗、干细胞治疗、饮食、营养补充剂、多学科诊所以及机械性有创和无创通气。本文还讨论了有关马西替尼、肠道喂养的作用、基因治疗和干细胞治疗的最新数据。
Maximizing the Survival of Amyotrophic Lateral Sclerosis Patients: Current Perspectives.
Amyotrophic lateral sclerosis is a neurodegenerative disease that leads to loss of the upper and lower motor neurons. Almost 90% of all cases occur in the sporadic form, with the rest occurring in the familial form. The disease has a poor prognosis, with only two disease-modifying drugs approved by the United States Food and Drug Administration (FDA). The approved drugs for the disease have very limited survival benefits. Edaravone is a new FDA-approved medication that may slow the disease progression by 33% in a selected subgroup of ALS patients. This paper covers the various interventions that may provide survival benefits, such as early diagnosis, medications, gene therapy, stem cell therapy, diet, nutritional supplements, multidisciplinary clinics, and mechanical invasive and noninvasive ventilation. The recent data on masitinib, the role of enteral feeding, gene therapy, and stem cell therapy is discussed.
期刊介绍:
Neurology Research International is a peer-reviewed, Open Access journal that publishes original research articles, review articles, and clinical studies focusing on diseases of the nervous system, as well as normal neurological functioning. The journal will consider basic, translational, and clinical research, including animal models and clinical trials.