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尼古拉德斯-巴拉伊泽综合征:表型描述

Nicolaides-Baraitser syndrome: Delineation of the phenotype.

作者信息

Sousa Sérgio B, Abdul-Rahman Omar A, Bottani Armand, Cormier-Daire Valérie, Fryer Alan, Gillessen-Kaesbach Gabriele, Horn Denise, Josifova Dragana, Kuechler Alma, Lees Melissa, MacDermot Kay, Magee Alex, Morice-Picard Fanny, Rosser Elizabeth, Sarkar Ajoy, Shannon Nora, Stolte-Dijkstra Irene, Verloes Alain, Wakeling Emma, Wilson Louise, Hennekam Raoul C M

机构信息

Department of Clinical Genetics, Great Ormond Street Hospital for Children, London, UK.

出版信息

Am J Med Genet A. 2009 Aug;149A(8):1628-40. doi: 10.1002/ajmg.a.32956.

Abstract

Nicolaides-Baraitser syndrome (NBS) is an infrequently described condition, thus far reported in five cases. In order to delineate the phenotype and its natural history in more detail, we gathered data on 18 hitherto unreported patients through a multi-center collaborative study, and follow-up data of the earlier reported patients. A detailed comparison of the 23 patients is provided. NBS is a distinct and recognizable entity, and probably has been underdiagnosed until now. Main clinical features are severe mental retardation with absent or limited speech, seizures, short stature, sparse hair, typical facial characteristics, brachydactyly, prominent finger joints and broad distal phalanges. Some of the features are progressive with time. The main differential diagnosis is Coffin-Siris syndrome. There is no important gender difference in occurrence and frequency of the syndrome, and all cases have been sporadic thus far. Microarray analysis performed in 14 of the patients gave normal results. Except for the progressive nature there are no clues to the cause.

摘要

尼古拉德斯 - 巴拉伊泽综合征(NBS)是一种较少被描述的病症,迄今为止仅报道过5例。为了更详细地描述其表型及自然病史,我们通过一项多中心协作研究收集了18例此前未报道患者的数据,以及早期报道患者的随访数据。对这23例患者进行了详细比较。NBS是一个独特且可识别的实体,可能在此之前一直未得到充分诊断。主要临床特征包括严重智力发育迟缓、言语缺失或受限、癫痫发作、身材矮小、头发稀疏、典型面部特征、短指畸形、指关节突出以及远端指骨宽阔。部分特征会随时间进展。主要鉴别诊断为科芬 - 西里斯综合征。该综合征的发生及频率在性别上无显著差异,迄今为止所有病例均为散发性。对14例患者进行的微阵列分析结果正常。除了其进展性外,尚无病因线索。

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