脊髓小脑共济失调 27B 型(SCA27B):葡萄牙的一种遗传性共济失调。

IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Acta medica portuguesa Pub Date : 2024-10-11 DOI:10.20344/amp.22232
Vítor Mendes Ferreira, Marta Magriço, Bruna Meira, Raquel Barbosa
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引用次数: 0

摘要

脊髓小脑共济失调 27B 型(SCA27B)是最近发现的一种遗传性疾病,由成纤维细胞生长因子 14(FGF14)基因中的(GAA)≥250 重复扩增引起,多项研究已确认它是欧洲人群中常染色体显性共济失调最常见的病因之一。我们报告了一例 62 岁的葡萄牙患者的病例,该患者出现缓慢进行性步态障碍,伴有宽基共济失调步态、构音障碍、左上肢构音障碍和运动障碍。这种纯粹的小脑表型会在剧烈运动和饮酒后发作性恶化。患者的哥哥患有晚发型小脑共济失调症,病因不明。基因检测发现了杂合子内含子 FGF14 重复扩增,且具有完全渗透性(> 360 个重复),从而确诊为 SCA27B。据我们所知,这是葡萄牙人群中报告的首例SCA27B病例。
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Spinocerebellar Ataxia Type 27B (SCA27B): A Hereditary Ataxia in Portugal.

Spinocerebellar ataxia type 27B (SCA27B) is a recently discovered hereditary disease caused by (GAA)≥250 repeat expansion in the fibroblast growth factor 14 (FGF14) gene, and multiple studies have recognized it as one of the most common causes of autosomal dominant ataxia in the European population. We present the case of a 62-year-old Portuguese patient who developed a slowly progressive gait impairment associated with wide-base ataxic gait, dysarthria, left upper limb dysmetria, and dysdiadochokinesia. This pure cerebellar phenotype had an episodic worsening induced by intense physical activity and alcohol intake. The patient had an older brother with a late-onset cerebellar ataxia of unknown cause. Genetic testing detected a heterozygotic intronic FGF14 repeat expansion with complete penetrance (> 360 repeats), confirming the diagnosis of SCA27B. To our knowledge, we present the first reported case of SCA27B in the Portuguese population.

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来源期刊
Acta medica portuguesa
Acta medica portuguesa MEDICINE, GENERAL & INTERNAL-
CiteScore
1.90
自引率
16.70%
发文量
256
审稿时长
6-12 weeks
期刊介绍: The aim of Acta Médica Portuguesa is to publish original research and review articles in biomedical areas of the highest standard, covering several domains of medical knowledge, with the purpose to help doctors improve medical care. In order to accomplish these aims, Acta Médica Portuguesa publishes original articles, review articles, case reports and editorials, among others, with a focus on clinical, scientific, social, political and economic factors affecting health. Acta Médica Portuguesa will be happy to consider manuscripts for publication from authors anywhere in the world.
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