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韦海伊综合征病例报告

A Case Report of Verheij Syndrome.

作者信息

Sivasubramanian Dhiran, Ayyavoo Ahila

机构信息

Critical Care Medicine, Christian Medical College, Vellore, IND.

Pediatric Endocrinology, G. Kuppuswamy Naidu Memorial Hospital, Coimbatore, IND.

出版信息

Cureus. 2024 Aug 12;16(8):e66692. doi: 10.7759/cureus.66692. eCollection 2024 Aug.

Abstract

Verheij syndrome (VRJS) is a rare genetic disorder characterized by a range of developmental issues and physical abnormalities. This condition is caused by mutations or deletions in the PUF60 (poly-U-binding factor 60 kDa) gene, which is located on the long arm of chromosome 8, specifically in the q24.3 region. We present a unique case of an 11-year-old girl child with VRJS. The child presented with absence seizures. She was noted to have short stature, spina bifida of the lower cervical vertebrae, and a smaller right kidney on ultrasonography. This case expands the phenotypic spectrum of VRJS by demonstrating a milder presentation, highlighting the importance of a high index of suspicion for the diagnosis, even in atypical presentations. Whole exome sequencing identified the causative mutation, confirming the diagnosis. Growth hormone therapy was initiated for short stature but discontinued due to the subsequent development of idiopathic intracranial hypertension. Additionally, this report represents the first documented case of VRJS in India, emphasizing the importance of global data sharing and collaboration for improving the understanding and management of rare genetic disorders.

摘要

韦赫伊综合征(VRJS)是一种罕见的遗传性疾病,其特征为一系列发育问题和身体异常。这种病症是由位于8号染色体长臂,具体为q24.3区域的PUF60(多聚-U结合因子60 kDa)基因突变或缺失引起的。我们报告了一例11岁患韦赫伊综合征女童的独特病例。该患儿出现失神发作。超声检查发现她身材矮小、下颈椎脊柱裂以及右肾较小。该病例通过展示较轻微的临床表现扩展了韦赫伊综合征的表型谱,强调了即使在非典型表现中,高度怀疑诊断的重要性。全外显子测序确定了致病突变,从而确诊。因身材矮小开始了生长激素治疗,但由于随后出现特发性颅内高压而停药。此外,本报告是印度首例记录在案的韦赫伊综合征病例,强调了全球数据共享与合作对于增进对罕见遗传疾病的理解和管理的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7f71/11390954/f8fea15d30fd/cureus-0016-00000066692-i01.jpg

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