遗传代谢性疾病的临床和生化足迹:XVII。先天性畸形。

IF 3.7 2区 生物学 Q2 ENDOCRINOLOGY & METABOLISM
Molecular genetics and metabolism Pub Date : 2025-01-01 Epub Date: 2024-12-13 DOI:10.1016/j.ymgme.2024.109001
Carol L Greene, Sofia Saenz-Ayala, Erin T Strovel, Francis Rossignol, Carlos R Ferreira, Nenad Blau
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引用次数: 0

摘要

畸形,或外观上的生理异常,在遗传代谢紊乱(IMD)的个体中可能在频率和严重程度上有所不同。这些疾病中畸形的患病率可以从罕见的情况到更常见的特征,这取决于具体的疾病及其遗传特征。值得注意的是,并非所有患有imd的个体都会表现出畸形特征,并且这些特征的存在在不同类型的代谢紊乱中可能存在很大差异。本研究中提供的数据包括374个具有畸形特征的imd的详细列表,按受影响的器官(如头和脸、鼻子、嘴和舌头、眼睛、耳朵、手和脚等)分类,以及重要临床特征的概述和推荐的诊断策略,对医疗保健领域的专业人员可能很有价值。这些信息对于治疗代谢紊乱个体的医疗保健提供者或那些护理表现出可能表明存在遗传性代谢紊乱的畸形特征的个体的医疗保健提供者可能特别有用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Clinical and biochemical footprints of inherited metabolic diseases: XVII. Dysmorphisms.

Dysmorphisms, or physical abnormalities in appearance, can vary in frequency and severity among individuals with inherited metabolic disorders (IMD). The prevalence of dysmorphisms in these disorders can range from rare occurrences to more common features, depending on the specific disorder and its genetic characteristics. It is important to note that not all individuals with IMDs will exhibit dysmorphic features, and the presence of such features may vary widely among different types of metabolic disorders. The data presented in this study, which includes a detailed list of 374 IMDs with dysmorphic characteristics categorized by affected organs (such as head and face, nose, mouth and tongue, eye, ear, hands and feet, and others), as well as an overview of important clinical features and recommended diagnostic strategies, could be valuable for professionals in the field of healthcare. This information may be particularly useful for healthcare providers who treat individuals with metabolic disorders or those who care for individuals exhibiting dysmorphic features that could indicate the presence of an inherited metabolic disorder.

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来源期刊
Molecular genetics and metabolism
Molecular genetics and metabolism 生物-生化与分子生物学
CiteScore
5.90
自引率
7.90%
发文量
621
审稿时长
34 days
期刊介绍: Molecular Genetics and Metabolism contributes to the understanding of the metabolic and molecular basis of disease. This peer reviewed journal publishes articles describing investigations that use the tools of biochemical genetics and molecular genetics for studies of normal and disease states in humans and animal models.
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