Asmus Friedrich, Zimprich Alexander, Tezenas Du Montcel Sophie, Kabus Christian, Deuschl Günther, Kupsch Andreas, Ziemann Ulf, Castro Mirna, Kühn Andrea A, Strom Tim M, Vidailhet Marie, Bhatia Kailash P, Dürr Alexandra, Wood Nicholas W, Brice Alexis, Gasser Thomas
Neurologische Klinik Grosshadern, Ludwig-Maximilians-Universität, Munich, Germany.
Ann Neurol. 2002 Oct;52(4):489-92. doi: 10.1002/ana.10325.
Mutations in the gene for epsilon-sarcoglycan (SGCE) have been found to cause myoclonus-dystonia syndrome. We now report clinical and genetic findings in nine additional European families with myoclonus-dystonia syndrome. The clinical presentation in 24 affecteds was homogeneous with myoclonus predominantly of neck and upper limbs in 23 of them and dystonia, presenting as cervical dystonia and/or writer's cramp, in 13 cases. Six novel and one previously known heterozygous SGCE mutations were identified. SGCE deficiency seems to be the common pathogenetic mechanism in myoclonus-dystonia syndrome.
已发现ε-肌聚糖(SGCE)基因的突变会导致肌阵挛性肌张力障碍综合征。我们现在报告另外9个患有肌阵挛性肌张力障碍综合征的欧洲家族的临床和基因研究结果。24名患者的临床表现具有同质性,其中23人主要表现为颈部和上肢的肌阵挛,13例出现肌张力障碍,表现为颈部肌张力障碍和/或书写痉挛。鉴定出6个新的和1个先前已知的杂合SGCE突变。SGCE缺乏似乎是肌阵挛性肌张力障碍综合征常见的发病机制。