Chinese Journal of Practical Pediatrics

Chinese Journal of Practical Pediatrics ›› 2022, Vol. 37 ›› Issue (4): 265-268.DOI: 10.19538/j.ek2022040606

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Multidisciplinary management and diagnosis and treatment mode of spinal muscular atrophy in China

  

  1. Department of Neurology，Children’s Hospital of Fudan University，National Children’s Medical Center，Shanghai  201102，China
  • Online:2022-04-06 Published:2022-05-23

我国脊髓性肌萎缩症多学科管理和诊治模式

  

  1. 复旦大学附属儿科医院神经科 国家儿童医学中心（上海），上海  201102
  • 通讯作者: 王艺，电子信箱：yiwang@shmu.edu.cn
  • 基金资助:
    复旦大学附属儿科医院重点发展项目（EK2022ZX01；EK2022ZX06）

Abstract: Spinal muscular atrophy（SMA）is characterized by degeneration of motor neurons in the spinal cord with progressive muscle atrophy，weakness and paralysis.Effective and efficient management of the patient with SMA requires coordination of multiple clinical specialists.The need for an update on multidisciplinary management of SMA has been driven by the approval of the drugs which are disease modifying therapy for SMA in China.The article aims to review and introduce multidisciplinary management of SMA.

Key words: spinal muscular atrophy, multidisciplinary management, disease modifying therapy

摘要： 脊髓性肌萎缩症（SMA）主要累及运动神经元导致肌无力及肌萎缩，在疾病不同阶段可出现多系统（骨骼、呼吸、消化等）合并症。随着SMA的疾病修正治疗药物在国内的应用及研究的开展，SMA多学科管理及诊治模式迎来了新的挑战。该文从疾病修正药物治疗、康复管理、骨骼管理、营养管理、呼吸管理等方面进行了介绍。

关键词: 脊髓性肌萎缩症, 多学科管理, 疾病修正治疗