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埃默里-德赖富斯肌营养不良症

Emery-Dreifuss muscular dystrophy.

作者信息

Zacharias A S, Wagener M E, Warren S T, Hopkins L C

机构信息

Department of Neurology, Emory University School of Medicine, Atlanta, Georgia 30322, USA.

出版信息

Semin Neurol. 1999;19(1):67-79. doi: 10.1055/s-2008-1040827.

Abstract

Emery-Dreifuss muscular dystrophy (EDMD) is the third most common X-linked muscular dystrophy. This disorder is characterized by childhood onset of early contractures, humeroperoneal muscle atrophy, and cardiac conduction abnormalities. Weakness is slowly progressive, but there is a broad spectrum of clinical severity. Patients and carriers are at risk of sudden death. Regular cardiac evaluation is mandatory to assess the risk of cardiac arrhythmias. Unique atrial pathology is seen at autopsy. The mutated gene in EDMD is localized to the long arm of the X chromosome. Mutations in the gene lead to abolished synthesis of the gene product, emerin. Emerin is localized to the nuclear membrane of skeletal, cardiac, and smooth muscle. The term Emery-Dreifuss syndrome describes patients who have the EDMD phenotype without X-linked inheritance. There is no treatment for the underlying disease, but early placement of pacemakers may be lifesaving.

摘要

埃默里-德赖富斯肌营养不良症(EDMD)是第三常见的X连锁肌营养不良症。这种疾病的特征是儿童期发病,伴有早期挛缩、肱腓肌萎缩和心脏传导异常。肌无力呈缓慢进行性发展,但临床严重程度差异很大。患者和携带者有猝死风险。必须定期进行心脏评估以评估心律失常的风险。尸检时可见独特的心房病理改变。EDMD中的突变基因定位于X染色体长臂。该基因突变导致基因产物emerin的合成被阻断。Emerin定位于骨骼肌、心肌和平滑肌的核膜。埃默里-德赖富斯综合征一词用于描述具有EDMD表型但无X连锁遗传的患者。目前尚无针对该基础疾病的治疗方法,但早期植入起搏器可能挽救生命。

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