Department of Child and Adolescent Neurology, Consultation of Movement Disorders and Botulinum Toxin, National Institute of Neurology, Tunis, Tunisia.
Eur J Paediatr Neurol. 2010 Jul;14(4):304-7. doi: 10.1016/j.ejpn.2009.11.005. Epub 2009 Dec 16.
Inborn errors of metabolism (IEM) are a group of genetic disorders characterized by dysfunction of an enzyme or other protein involved in cellular metabolism.(1) Most IEMs involve the nervous system (neuro-metabolic diseases or NMD). NMD often present with a complex clinical picture: psychomotor retardation and/or regression, pyramidal signs, ataxia, hypotonia and epilepsy and movement disorders.(1) Movement disorders are more frequently part of this complex picture than a predominant symptom, however in some instances the clinical picture may be summarized in an invalidating movement disorder.(2) On a phenomenology basis, one can distinguish eight main types of movement disorders: dystonia and athetosis, chorea, tremor with or without parkinsonism, ballismus, myoclonus, tics and stereotypies. Most of these abnormal involuntary movements generate from a dysfunction or a lesion in the basal ganglia, excepting myoclonus, the origin of which can vary (cortical, brainstem, basal ganglia, spinal and even peripheral nervous system).(3) Classically the most frequently observed movement disorders in NMD are: dystonia, myoclonus, chorea, tremor and parkinsonism (Fig. 1). The primary goal of this article is, departing from the literature and a large personal series, to describe the types of movement disorders most frequently observed in NMD and to discuss their clinical value in the setting of specific types of NMD.
先天性代谢缺陷(IEM)是一组以参与细胞代谢的酶或其他蛋白质功能障碍为特征的遗传疾病。(1)大多数 IEM 涉及神经系统(神经代谢疾病或 NMD)。NMD 常表现出复杂的临床特征:精神运动发育迟缓或倒退、锥体束征、共济失调、肌张力低下和癫痫发作以及运动障碍。(1)运动障碍在这种复杂的表现中更为常见,而不是主要症状,但在某些情况下,临床特征可能以致残性运动障碍来概括。(2)基于现象学,可将运动障碍分为八种主要类型:肌张力障碍和舞蹈手足徐动症、舞蹈症、震颤伴或不伴帕金森病、投掷症、肌阵挛、抽动和刻板动作。除了肌阵挛,这些异常不自主运动大多源自基底节的功能障碍或病变(皮质、脑干、基底节、脊髓甚至周围神经系统)。(3)经典的 NMD 中最常观察到的运动障碍是:肌张力障碍、肌阵挛、舞蹈症、震颤和帕金森病(图 1)。本文的主要目的是从文献和大量个人系列中描述在 NMD 中最常观察到的运动障碍类型,并讨论它们在特定类型的 NMD 中的临床价值。