Chinese Journal of Contemporary Neurology and Neurosurgery ›› 2018, Vol. 18 ›› Issue (4): 284-289. doi: 10.3969/j.issn.1672-6731.2018.04.010

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Research progress of spinal muscular atrophy treatment in children

ZHAO Miao, LU Ying-qian, WANG Ning, CHEN Wan-jin   

  1. Department of Neurology, the First Affiliated Hospital of Fujian Medical University, Fuzhou 350005, Fujian, China
  • Online:2018-04-25 Published:2018-04-18
  • Contact: CHEN Wan-jin (Email: wanjinchen75@fjmu.edu.cn)
  • Supported by:

    This study was supported by the National Natural Science Foundation of China (No. U1505222, 81371261), Key Clinical Specialty Discipline Construction Program of Fujian, China, and National Key Clinical Specialty Discipline Construction Program on Neurology.

儿童型脊髓性肌萎缩症治疗研究进展

赵淼, 陆瑛倩, 王柠, 陈万金   

  1. 350005 福州,福建医科大学附属第一医院神经内科
  • 通讯作者: 陈万金(Email:wanjinchen75@fjmu.edu.cn)
  • 基金资助:

    国家自然科学基金资助项目(项目编号:U1505222);国家自然科学基金资助项目(项目编号:81371261);福建省临床重点专科建设项目;神经内科国家临床重点专科建设项目

Abstract:

Spinal muscular atrophy (SMA) is the most common fatal neurogenetic disease in infant period. Clinical manifestations of SMA include symmetrical and progressive weakness and atrophy of proximal limbs. Based on age of incidence, it is divided into child-onset SMA and adult-onset SMA. Child-onset SMA has the highest incidence rate. This article summarizes the research progress of SMA therapy in recent years to provide new thoughts for the treatment of SMA.

Key words: Spinal muscular atrophies of childhood, Genes, Review

摘要:

脊髓性肌萎缩症是婴幼儿期最常见的致死性神经遗传性疾病,临床表现为肢体近端进行性、对称性肌无力和肌萎缩。根据发病年龄可以分为儿童型和成人型,尤以儿童型脊髓性肌萎缩症发病率最高。本文总结近年儿童型脊髓性肌萎缩症的治疗研究进展,旨在为疾病治疗提供新的思路。

关键词: 脊髓性肌萎缩, 儿童, 基因, 综述