Neurology Deparment, Centro Hospitalar e Universitario de Coimbra EPE, Coimbra, Portugal
Neurology Deparment, Centro Hospitalar e Universitario de Coimbra EPE, Coimbra, Portugal.
BMJ Case Rep. 2022 Nov 15;15(11):e245764. doi: 10.1136/bcr-2021-245764.
Familial amyloidosis of the Finnish type (FAF) is a rare multisystemic disorder caused by mutations in the gelsolin gene. The clinical presentation is typically characterised by a triad of ophthalmic, neurological and dermatological findings. FAF has been reported in several countries, primarily in Finland and recently in Portugal. We report the first genetically confirmed cases of FAF from two unrelated families in our neuromuscular outpatient clinic. Gelsolin gene sequencing revealed the heterozygous gelsolin mutation (c.640G>A). The clinical features and the neurophysiological studies of two index patients and their relatives are presented. Obtaining an early diagnosis can be challenging, but FAF should be considered in the differential diagnosis of progressive bilateral facial neuropathy, even if there is no known Finnish ancestor.
芬兰型家族性淀粉样变性(FAF)是一种罕见的多系统疾病,由凝胶蛋白基因突变引起。其临床表现通常以眼科、神经和皮肤三联征为特征。FAF 已在多个国家报告,主要在芬兰,最近在葡萄牙也有报告。我们报告了首例来自两个无血缘关系的神经肌肉门诊患者家系的 FAF 基因确诊病例。凝胶蛋白基因突变测序发现杂合性凝胶蛋白突变(c.640G>A)。本文呈现了两位先证者及其亲属的临床特征和神经生理学研究。早期诊断可能具有挑战性,但即使没有已知的芬兰血统,也应考虑 FAF 作为进行性双侧面神经病的鉴别诊断。