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两例新病例具有反映 Schaaf-Yang 综合征临床多样性的新型致病性变异。

Two new cases with novel pathogenic variants reflecting the clinical diversity of Schaaf-Yang syndrome.

机构信息

Department of Medical Genetics, School of Medicine, Marmara University, Istanbul, Turkey.

Department of Medical Genetics, Istanbul University-Cerrahpaşa, Cerrahpaşa Faculty of Medicine, Istanbul, Turkey.

出版信息

Clin Genet. 2023 Jul;104(1):127-132. doi: 10.1111/cge.14320. Epub 2023 Mar 9.

Abstract

Schaaf-Yang syndrome (SHFYNG) is a rare pleiotropic disorder, characterized by hypotonia, joint contractures, autism spectrum disorders (ASD), and developmental delay/intellectual disability. Although it shares some common features with Prader-Willi Syndrome, joint contractures, and ASD were more commonly detected in in this syndrome. Recently, it was shown that truncating variants in the paternal allele of the MAGEL2 gene cause SHFYNG. Here, we present two patients diagnosed with SHFYNG syndrome having two different novel truncating variants in the MAGEL2 gene, one paternally inherited and one de novo. One patient had obesity, brachydactyly and dysmorphic features, and the other patient presented with contractures, severe hypotonia and early death. This is the first report of Turkish SHFYNG syndrome cases presented to emphasize the phenotypic diversity of the syndrome.

摘要

Schaaf-Yang 综合征(SHFYNG)是一种罕见的多系统疾病,其特征为张力减退、关节挛缩、自闭症谱系障碍(ASD)和发育迟缓/智力残疾。虽然它与 Prader-Willi 综合征有一些共同特征,但在该综合征中更常检测到关节挛缩和 ASD。最近,研究表明 MAGEL2 基因父源等位基因的截断变异导致了 SHFYNG。在这里,我们介绍了两位被诊断为 Schaaf-Yang 综合征的患者,他们在 MAGEL2 基因中分别携带一个父源遗传和一个新生的不同的截断变异。一位患者存在肥胖、短指畸形和畸形特征,另一位患者表现为挛缩、严重张力减退和早亡。这是首例报道的土耳其 Schaaf-Yang 综合征病例,旨在强调该综合征的表型多样性。

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