Suppr超能文献

伴有韦斯特综合征的严重早发型乙基丙二酸脑病

Severe early onset ethylmalonic encephalopathy with West syndrome.

作者信息

Papetti Laura, Garone Giacomo, Schettini Livia, Giordano Carla, Nicita Francesco, Papoff Paola, Zeviani Massimo, Leuzzi Vincenzo, Spalice Alberto

机构信息

Division of Child Neurology, Department of Paediatrics, Sapienza University of Rome, Rome, Italy.

Department of Radiological, Oncological and Pathological Sciences, Sapienza University of Rome, Rome, Italy.

出版信息

Metab Brain Dis. 2015 Dec;30(6):1537-45. doi: 10.1007/s11011-015-9707-8. Epub 2015 Jul 21.

Abstract

Ethylmalonic encephalopathy (EE) is a rare autosomal recessive disorder characterized by early onset encephalopathy, chronic diarrhoea, petechiae, orthostatic acrocyanosis and defective cytochrome c oxidase (COX) in muscle and brain. High levels of lactic, ethylmalonic and methylsuccinic acids are detected in body fluids. EE is caused by mutations in ETHE1 gene, a mitochondrial sulfur dioxygenase. Neurologic signs and symptoms include progressively delayed development, hypotonia, seizures, and abnormal movements. We report on the clinical, electroencephalographic and MRI findings of a baby with a severe early onset encephalopathy associated with novel ETHE1 gene mutation. This is the first case described in literature with an early pure epileptic onset, presenting with West syndrome.

摘要

乙基丙二酸脑病(EE)是一种罕见的常染色体隐性疾病,其特征为早发性脑病、慢性腹泻、瘀点、体位性手足发绀以及肌肉和大脑中的细胞色素c氧化酶(COX)缺陷。在体液中检测到高水平的乳酸、乙基丙二酸和甲基琥珀酸。EE由线粒体硫双加氧酶ETHE1基因突变引起。神经学体征和症状包括发育逐渐延迟、肌张力减退、癫痫发作和异常运动。我们报告了一名患有严重早发性脑病并伴有新型ETHE1基因突变的婴儿的临床、脑电图和磁共振成像结果。这是文献中描述的首例以早期单纯癫痫发作为表现、呈现韦斯特综合征的病例。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验