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描述一名年轻男孩出现一种特殊的交替发作性电临床模式,该男孩携带一种新型的 SPATA5 突变。

Description of a peculiar alternating ictal electroclinical pattern in a young boy with a novel SPATA5 mutation.

机构信息

Institute for Maternal and Child Health, IRCCS "Burlo Garofolo", Trieste.

Child Neurology and Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bologna.

出版信息

Epileptic Disord. 2020 Oct 1;22(5):659-663. doi: 10.1684/epd.2020.1204.

Abstract

Heterozygous variants in the SPATA5 gene have recently been described to be associated with epileptic encephalopathy. As of 2019, 37 patients have been described in the published literature. We report a patient with a novel autosomal recessive pathogenic variant in SPATA5 and a clinical phenotype consistent with SPATA5 syndrome, including severe neurological impairment, intellectual disability (ID), generalized intractable epilepsy, microcephaly, abnormal muscle tone, and sensorineural hearing loss. The epileptic clinical features were characterized by infantile spasms associated with seizures with a complex ocular movement; a predominant involvement of the posterior cerebral area and cortical visual impairment were also noticed. This phenotype is highlighted with a review of the literature showing other patients with SPATA5-related disease. This report aims to contribute to further understanding phenotype/genotype correlations, which are fundamental for the interpretation of data made available by exome sequencing for the diagnosis of epileptic encephalopathies. [Published with video sequence].

摘要

SPATA5 基因中的杂合变异最近被描述与癫痫性脑病有关。截至 2019 年,已在已发表的文献中描述了 37 例患者。我们报告了一例 SPATA5 中新型常染色体隐性致病性变异的患者,其临床表现与 SPATA5 综合征一致,包括严重的神经功能障碍、智力障碍(ID)、全身性难治性癫痫、小头畸形、肌肉张力异常和感觉神经性听力损失。癫痫的临床特征表现为伴有复杂眼球运动的婴儿痉挛症相关发作;还注意到后脑区和皮质视觉损害的主要受累。这一表型通过文献回顾得到了强调,其中包括其他具有 SPATA5 相关疾病的患者。本报告旨在进一步了解表型/基因型相关性,这对于解释外显子组测序提供的用于诊断癫痫性脑病的数据至关重要。[随附视频序列发布]。

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