文献类型：期刊文献

中文题名：脊髓性肌萎缩症最新药物治疗及用药护理进展

英文题名：Advances in the latest drug treatment and medication care for spinal muscular atrophy

作者：王钰艳[1];王金霞[1];吴桃民[1,2];石雨轩[1]

第一作者：王钰艳

机构：[1]甘肃中医药大学护理学院,甘肃兰州730000;[2]甘肃省中心医院神经外科,甘肃兰州730050

第一机构：甘肃中医药大学护理学院

年份：2026

卷号：42

期号：4

起止页码：880

中文期刊名：现代医药卫生

外文期刊名：Journal of Modern Medicine & Health

语种：中文

中文关键词：脊髓性肌萎缩症;药物治疗;不良反应;护理;综述

外文关键词：Spinal muscular atrophy;Drug therapy;Adverse reactions;Nursing;Review

摘要：脊髓性肌萎缩症(SMA)是一种罕见的脊髓前角运动神经元变性导致的常染色体隐性遗传的退行性神经肌肉疾病,主要临床表现为近端肢体和躯干对称性肌无力和肌萎缩,可致死、致残。近年来,随着SMA修正治疗药物的不断出现,能有效延缓SMA疾病进展、减少患者肌肉萎缩或畸形、改善其生活质量。由于该病较为罕见,临床中修正药物的使用频率较低,有关此类药物的长期有效性、安全性及用药护理等方面的报道相对较少,因此仍需开展进一步的监测与研究。该文通过对治疗SMA的修正治疗药物及用药护理进行综述,以期对SMA精准化及个体化治疗提供参考依据。
Spinal muscular atrophy(SMA)is a rare degenerative neuromuscular disease caused by the degeneration of anterior horn motor neurons in the spinal cord,with an autosomal recessive inheritance pattern.Its main clinical manifestations include symmetric muscle weakness and atrophy in the proximal limbs and trunk,which can lead to disability or even death.In recent years,with the continuous emergence of disease-modifying therapies for SMA,these treatments have been effective in slowing down disease progression,reducing muscle atrophy or deformities,and improving patients'quality of life.However,due to the rarity of the disease,the clinical application of such disease-modifying therapies remains relatively infrequent,and there is a lack of reports on their long-term efficacy,safety,and medication care.Therefore,further monitoring and research are still needed.This article reviewed the disease-modifying therapies for SMA and related medication care,aiming to provide a reference for the precise and individualized treatment of SMA.