Research progress on biomarkers for the monitoring of Spinal muscular atrophy
翦烨敏 1吴丽文 2鞠翠钰
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作者信息
1. 南华大学衡阳医学院，南华大学儿科学院（湖南省儿童医院）神经内科，衡阳 421001
2. 湖南省儿童医院神经内科，长沙 410007
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摘要
脊髓性肌萎缩(SMA)是儿童最常见的神经肌肉病，严重影响儿童的生命健康。目前SMA的基因和分子修饰治疗已成为热点，然而SMA患者何时开始治疗、治疗效果以及疾病预后都有许多不确定性，因此迫切需要可靠的生物标志物进行监测和评估。本文将近年来SMA生物标志物研究取得的进展进行综述。 Spinal muscular atrophy (SMA) is the most common neuromuscular disease in children, which seriously affects children′s health. At present, gene and molecular modification therapy for SMA have become hot spots. However, there are many uncertainties about when people with SMA should start treatment, how well the drugs can treat, and the prognosis. Therefore, reliable biomarkers for monitoring and evaluation are urgently needed. This review will summarize the progress made in SMA biomarker research in recent years.
Abstract
Spinal muscular atrophy (SMA) is the most common neuromuscular disease in children, which seriously affects children′s health. At present, gene and molecular modification therapy for SMA have become hot spots. However, there are many uncertainties about when people with SMA should start treatment, how well the drugs can treat, and the prognosis. Therefore, reliable biomarkers for monitoring and evaluation are urgently needed. This review will summarize the progress made in SMA biomarker research in recent years.
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