Contarino Maria Fiorella, Berger-Plantinga Elles, Foncke Elisabeth M J, Ritz Katja, Mellema Jonke, Baas Frank, Speelman Johannes D, Tijssen Marina A J
Department of Neurology and Clinical Neurophysiology, Academic Medical Centre, Amsterdam, The Netherlands.
Mov Disord. 2008 Oct 30;23(14):1998-2003. doi: 10.1002/mds.22206.
We describe a large family with a primary focal dystonia from a small Dutch village on a former island. Twenty-four individuals spanning three generations were examined by two movement-disorder neurologists. Two other movement-disorder neurologists evaluated the videos independently. Subjects were classified as "affected," "possibly affected," or "not affected." A diagnosis was defined if all the neurologists agreed on the definition. Eight definitely affected and four possibly affected subjects were detected. Clinical presentation consisted of mild cranio-cervical-brachial dystonia. Mean age at onset was 45.5 years (range, 39-56). Mean BFMDRS motor score was 4.4 (range, 1-8). Mean TWSTRS score (part I) was 11.3 (range, 8-23). Mutations in DYT1 gene and in the epsilon-sarcoglycan (SGCE) genes were not detected. We could not find linkage to the dominant DYT6, DYT7, DYT13, or the recessive DYT16 loci. The identification and accurate clinical evaluation of large dystonia families not linked to known genes is crucial for further advancement in molecular genetic characterization of focal dystonia.
我们描述了一个来自前岛屿上一个荷兰小村庄的原发性局灶性肌张力障碍的大家族。两位运动障碍神经科医生对三代中的24名个体进行了检查。另外两位运动障碍神经科医生独立评估了视频。受试者被分类为“受影响”、“可能受影响”或“未受影响”。如果所有神经科医生对定义达成一致,则做出诊断。检测到8名明确受影响和4名可能受影响的受试者。临床表现为轻度颅颈臂肌张力障碍。发病的平均年龄为45.5岁(范围39 - 56岁)。BFMDRS运动评分平均为4.4(范围1 - 8)。TWSTRS评分(第一部分)平均为11.3(范围8 - 23)。未检测到DYT1基因和ε - 肌聚糖(SGCE)基因的突变。我们未发现与显性DYT6、DYT7、DYT13或隐性DYT16位点的连锁关系。对与已知基因无连锁关系的大型肌张力障碍家族进行识别和准确的临床评估对于局灶性肌张力障碍分子遗传学特征的进一步进展至关重要。