Giovanniello T, Leuzzi V, Carducci C, Carducci C, Sabato M L Di, Artiola C, Santagata S, Pozzessere S, Antonozzi I
Department of Experimental Medicine and Pathology, University La Sapienza, Rome, Italy.
Neuropediatrics. 2007 Aug;38(4):213-5. doi: 10.1055/s-2007-991151.
Tyrosine hydroxylase deficiency, a cause of the autosomal recessive form of L-DOPA responsive dystonia, has been associated with a broad spectrum of movement disorders and clinical courses. We describe a new patient presenting with an early onset spastic paraplegia who later developed a progressive generalized dystonic-dyskinetic syndrome. He markedly improved with a very low dosage of L-DOPA/carbidopa, while higher dosages were not tolerated. Two novel mutations (p.G414R/p.L510Q) were detected in the TH gene.
酪氨酸羟化酶缺乏症是常染色体隐性L-多巴反应性肌张力障碍的病因,与广泛的运动障碍和临床病程相关。我们描述了一名新患者,该患者最初表现为早发性痉挛性截瘫,后来发展为进行性全身性肌张力障碍-运动障碍综合征。他使用极低剂量的左旋多巴/卡比多巴后有明显改善,而高剂量则无法耐受。在TH基因中检测到两个新的突变(p.G414R/p.L510Q)。