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脑发育不全、神经病、鱼鳞病和皮肤角化病(CEDNIK)综合征的扩展表型谱:一例伴有室上性心动过速和脊髓栓系的罕见病例

Expanded Phenotypic Spectrum of Cerebral Dysgenesis, Neuropathy, Ichthyosis, and Keratoderma (CEDNIK) Syndrome: A Rare Case Featuring Supraventricular Tachycardia and Tethered Spinal Cord.

作者信息

Fuad Noor, Jadah Raafat Hamad Seroor H

机构信息

Medicine and Surgery, Bahrain Defence Force Hospital, Riffa, BHR.

Pediatric Neurology, Bahrain Defence Force Hospital, Riffa, BHR.

出版信息

Cureus. 2025 Jun 23;17(6):e86633. doi: 10.7759/cureus.86633. eCollection 2025 Jun.

Abstract

Cerebral dysgenesis, neuropathy, ichthyosis, and keratoderma (CEDNIK) syndrome is a rare, autosomal recessive neurocutaneous disorder. It represents a progressive neurodegenerative condition caused by mutations in the synaptosome-associated protein 29 (SNAP29) gene, which encodes a member of the soluble N-ethylmaleimide-sensitive factor attachment protein receptor (SNARE) family. This protein plays a critical role in intracellular membrane fusion and protein trafficking. Mutations in SNAP29 disrupt normal cellular processes, resulting in a broad spectrum of clinical manifestations, including facial dysmorphisms, microcephaly, severe developmental delay, hypotonia, ichthyosis, and peripheral neuropathy. In this report, we describe a rare case of CEDNIK syndrome featuring novel clinical findings, supraventricular tachycardia (SVT) and a tethered spinal cord, both of which have not been previously documented in association with this syndrome. These observations contribute to the expanding phenotypic spectrum of CEDNIK syndrome.

摘要

脑发育不全、神经病变、鱼鳞病和皮肤角化病(CEDNIK)综合征是一种罕见的常染色体隐性神经皮肤疾病。它是一种由突触体相关蛋白29(SNAP29)基因突变引起的进行性神经退行性疾病,该基因编码可溶性N - 乙基马来酰亚胺敏感因子附着蛋白受体(SNARE)家族的一个成员。这种蛋白质在细胞内膜融合和蛋白质运输中起关键作用。SNAP29基因的突变会破坏正常的细胞过程,导致广泛的临床表现,包括面部畸形、小头畸形、严重发育迟缓、肌张力减退、鱼鳞病和周围神经病变。在本报告中,我们描述了一例罕见的CEDNIK综合征病例,其具有新的临床发现,即室上性心动过速(SVT)和脊髓栓系,此前均未记录到该综合征伴有这两种情况。这些观察结果有助于扩大CEDNIK综合征的表型谱。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/50b0/12287679/8d652a484385/cureus-0017-00000086633-i01.jpg

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